19. ライソゾーム病 Disease details / Clinical trials / Drug dev / DR info

1- deoxygalactonojirimycin
   Amicus Therapeutics
      2005   Phase 2   EUCTR2005-004384-33-GB   United Kingdom
1-deoxygalactonojirimycin hydrochloride
   AMICUS THERAPEUTICS, INC
      2011   -   EUCTR2010-022636-37-IT   Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2012   -   EUCTR2010-022709-16-GB   Belgium;United Kingdom
      2012   -   EUCTR2010-022636-37-DE   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   Phase 2   EUCTR2010-022709-16-BE   Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom
      2011   Phase 3   EUCTR2010-022636-37-GR   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-GB   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-DK   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-BE   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-AT   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2010   Phase 3   EUCTR2009-013459-31-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2010   Phase 3   EUCTR2009-013459-31-ES   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2010   -   EUCTR2009-013459-31-DK   Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States
      2010   Phase 3   EUCTR2009-013459-31-BE   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 3   EUCTR2009-013459-31-GB   Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2009-013459-31-FR   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 2   EUCTR2007-001838-13-GB   Australia;Brazil;United Kingdom;United States
      2006   Phase 2   EUCTR2006-000181-36-GB   United Kingdom
      -   -   EUCTR2009-013459-31-DE   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
12 lead electrocardiogram
   Baylor Research Institute
      2018   -   NCT04002531   United States
12S-IT
   Shire HGT Inc
      2012   Phase 1;Phase 2   EUCTR2011-000212-25-GB   Canada;United Kingdom;United States
15-0 H20
   National Human Genome Research Institute (NHGRI)
      2006   -   NCT00302146   United States
1629063-78-0
   Genzyme Corporation
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-FR   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      -   Phase 2;Phase 3   EUCTR2020-003120-17-SE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2023   Phase 3   EUCTR2021-005402-10-IT   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002350-90-IT   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
   Sanofi-Aventis Recherche & Développement
      2022   Phase 3   EUCTR2021-002350-90-RO   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-PL   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-NO   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-FR   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-FI   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-AT   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2021   Phase 3   EUCTR2021-002350-90-GR   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DK   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DE   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
   Sanofi-aventis recherche & développement
      2022   Phase 3   EUCTR2021-005402-10-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005402-10-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Türkiye;United Kingdom;United States
   sanofi-aventis recherche et développement
      2022   Phase 3   EUCTR2021-002320-20-PL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NO   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-DK   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-CZ   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-AT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002320-20-GR   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-002320-20-ES   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
2(S)-(acetylamino)-4-methylpentanoic acid
   IntraBio Ltd
      2022   Phase 3   EUCTR2021-005356-10-SK   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-NL   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-DE   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-CZ   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004406-25-ES   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004406-25-DE   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-SK   Germany;Slovakia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-ES   Germany;Slovakia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-DE   Germany;Slovakia;Spain;United Kingdom;United States
2-aminoethanethiol
   Recordati Rare Diseases
      2021   Phase 3   EUCTR2018-002984-24-BE   Belgium;France;Germany;Italy;United Kingdom
      2020   Phase 3   EUCTR2018-002984-24-IT   Belgium;France;Germany;Italy;United Kingdom
      2020   Phase 3   EUCTR2018-002984-24-DE   France;Germany;United Kingdom
2-hour holter monitor
   Baylor Research Institute
      2018   -   NCT04002531   United States
2-hydroxypropyl-BETA-cyclodextrin
   Jichi Medical University, Saitama Medical Center
      2014   -   JPRN-UMIN000020032   Japan
   Nara Medical University
      2016   -   JPRN-UMIN000023517   Japan
      2014   -   JPRN-UMIN000023094   Japan
26 common fabry mutation types IN taiwan
   Chang Gung Memorial Hospital
      2016   -   NCT02859363   Taiwan
4D-310
   4D Molecular Therapeutics
      2022   Phase 1/Phase 2   NCT05629559   Australia;Taiwan
      2020   Phase 1/Phase 2   NCT04519749   United States
500 0500 0
   Manchester University NHS Foundation Trust
      2014   Phase 3   EUCTR2013-001479-18-GB   United Kingdom
68GA-dotatate PET/MRI
   University of Cambridge
      2024   -   NCT06226987   United Kingdom
89ZR-SBC-103
   Alexion Pharmaceuticals, Inc.
      2016   Phase 2   EUCTR2015-000753-20-NL   Netherlands
A16ax06
   Orphazyme ApS
      2015   Phase 1   EUCTR2014-005194-37-ES   Germany;Spain;Switzerland
      2015   Phase 1   EUCTR2014-005194-37-DE   Germany;Spain;Switzerland
AAV2/8.TBG.harsb
   FONDAZIONE TELETHON
      2018   Phase 1   EUCTR2016-002328-10-NL   Italy;Netherlands;Turkey
      2017   Phase 1   EUCTR2016-002328-10-IT   Italy;Netherlands;Turkey
   Fondazione Telethon
      2017   Phase 1/Phase 2   NCT03173521   Italy;Netherlands;Turkey
AAV9-CAG-COH-sgsh
   Laboratorios del Dr. Esteve, S.A.
      2016   Phase 1;Phase 2   EUCTR2015-000359-26-ES   Spain
AAV9-GLB1
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Aavrh.10-hmpsiiia
   SANFILIPPO Therapeutics SAS
      -   -   EUCTR2010-019962-10-FR   France
Aavrh.10cuarsa
   Inserm
      2012   Phase 1;Phase 2   EUCTR2011-004410-42-FR   France
Aavrh.10cucln2
   Weill Medical College of Cornell University
      2010   Phase 1/Phase 2   NCT01414985   United States
Aavrh.10cuhcln2 vector 2.85x10^11 genome copies
   Weill Medical College of Cornell University
      2010   Phase 1   NCT01161576   United States
Aavrh.10cuhcln2 vector 9.0x10^11 genome copies
   Weill Medical College of Cornell University
      2010   Phase 1   NCT01161576   United States
Abatacept
   Emory University
      2014   Phase 1   NCT01917708   United States
Abcertin
   ISU Abxis Co., Ltd.
      2020   Phase 1   NCT04787887   Australia
Abdominal ultrasound
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
ABO-101
   Abeona Therapeutics Europe SL.
      2021   Phase 1;Phase 2   EUCTR2019-002936-97-FR   France;Germany;United States
      2019   Phase 1;Phase 2   EUCTR2014-001411-39-GB   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2019   Phase 1;Phase 2   EUCTR2014-001411-39-DE   France;Germany;Spain;United Kingdom;United States
      -   Phase 1;Phase 2   EUCTR2019-002936-97-DE   France;Germany;Spain;United States
      -   Phase 1;Phase 2   EUCTR2014-001411-39-FR   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Abeona Therapeutics, Inc
      2020   -   NCT04655911   France;Germany;United States
ABO-102
   Abeona Therapeutics Inc
      2020   Phase 1;Phase 2   EUCTR2019-002979-34-ES   Australia;France;Germany;Spain;United States
      2019   Phase 1;Phase 2   EUCTR2018-000504-42-ES   Australia;Spain;United States
      2019   Phase 1;Phase 2   EUCTR2015-003904-21-FR   Australia;Brazil;France;Germany;Spain;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc
      2019   Phase 1/Phase 2   NCT04088734   Australia;Spain;United States
Accupro
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Accuretic
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Acetaminophen
   Sanofi
      2023   Phase 4   NCT06019728   United States
ACT-434964
   IDORSIA PHARMACEUTICALS LTD
      2020   Phase 3   EUCTR2018-002210-12-IT   Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-IT   Australia;Austria;Belgium;Canada;Finland;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
   Idorsia Pharmaceuticals Japan Ltd
      2019   Phase 3   JPRN-jRCT2080224659   Japan
   Idorsia Pharmaceuticals Ltd
      2021   Phase 3   EUCTR2018-002210-12-NO   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2021   Phase 3   EUCTR2018-002210-12-FR   Austria;Belgium;Canada;Czech Republic;France;Germany;Netherlands;Poland;United Kingdom;United States
      2020   Phase 3   EUCTR2018-002210-12-BE   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-NO   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-IE   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-ES   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Russian Federation;Spain;Switzerland;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-NL   Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-GB   Australia;Austria;Belgium;Canada;Germany;Ireland;Netherlands;Poland;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-DE   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-AT   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2017-003369-85-NL   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2018   Phase 3   EUCTR2017-003369-85-GB   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2018   Phase 3   EUCTR2017-003369-85-AT   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
Adalimumab
   Los Angeles Biomedical Research Institute
      2015   Phase 1/Phase 2   NCT02437253   United States
   Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
      2017   Phase 1/Phase 2   NCT03153319   United States
Adeno-associated viral (AAV) serotype 8 (AAV2/8) vector with liver-specific thyroxine-binding globulin (TBG) promoter, driving THE expression OF THE human arsb gene
   FONDAZIONE TELETHON
      2018   Phase 1   EUCTR2016-002328-10-NL   Italy;Netherlands;Turkey
Adeno-associated viral vector serotype 9 containing THE human N-acetyl-alpha-glucosaminidase gene
   Abeona Therapeutics Europe SL.
      2021   Phase 1;Phase 2   EUCTR2019-002936-97-FR   France;Germany;United States
      -   Phase 1;Phase 2   EUCTR2019-002936-97-DE   France;Germany;Spain;United States
Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cdna
   Sangamo Therapeutics, Inc.
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-IT   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-DE   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
      2019   Phase 1;Phase 2   EUCTR2019-000667-24-GB   United Kingdom;United States
Adeno-associated virus serotype 2/6 encoding human iduronate 2-sulfatase (hids) gene donor vector, SB-A6P-HNT donor vector
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000192-33-GB   United Kingdom;United States
Adeno-associated virus serotype 2/6 encoding left SIDE-zinc finger nuclease (ZFN1), SB-A6P-zleft vector
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000206-28-GB   United Kingdom;United States
      2018   Phase 1;Phase 2   EUCTR2018-000192-33-GB   United Kingdom;United States
Adeno-associated virus serotype 2/6 encoding right SIDE-zinc finger nuclease (ZFN2), SB-A6P-zright vector
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000206-28-GB   United Kingdom;United States
      2018   Phase 1;Phase 2   EUCTR2018-000192-33-GB   United Kingdom;United States
Adeno-associated virus serotype HU68 containing THE human galc gene
   Passage Bio, Inc.
      2021   Phase 1;Phase 2   EUCTR2020-005229-95-NL   Brazil;Canada;Israel;Netherlands;United Kingdom;United States
Adeno-associated virus serotype RH.10 expressing BETA-galactosidase
   Lysogene
      -   Phase 1;Phase 2   EUCTR2019-004949-32-FR   France;United Kingdom;United States
Adjuvant IM therapy
   Ultragenyx Pharmaceutical Inc
      2016   Phase 2/Phase 3   NCT02716246   Australia;France;Germany;Spain;United States
Adjuvant immunomodulatory (IM) therapy
   Ultragenyx Pharmaceutical Inc
      2020   Phase 3   NCT04360265   Australia;Spain;United States
Administration KIT
   BioMarin Pharmaceutical
      2020   -   NCT04476862   United States
Adrabetadex
   Mandos LLC
      2018   Phase 3   NCT03643562   United States
Afegostat tartrate
   Amicus Therapeutics
      2009   Phase 2   NCT00813865   Paraguay;United Kingdom;United States
      2008   Phase 2   NCT00446550   Germany;Israel;South Africa;United Kingdom;United States
      2007   Phase 2   NCT00433147   United States
   Amicus Therapeutics, Inc.
      2009   -   EUCTR2008-007158-36-GB   United Kingdom
Agalsidase
   Amicus Therapeutics
      2011   Phase 3   NCT01218659   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;Poland;Switzerland;Taiwan;Turkey;United Kingdom;United States
Agalsidase alfa
   AMICUS THERAPEUTICS, INC
      2011   -   EUCTR2010-022636-37-IT   Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States
   Amicus Therapeutics
      2011   Phase 2   NCT01196871   Australia;Belgium;Canada;France;Netherlands;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2012   -   EUCTR2010-022709-16-GB   Belgium;United Kingdom
      2012   -   EUCTR2010-022636-37-DE   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   Phase 2   EUCTR2010-022709-16-BE   Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom
      2011   Phase 3   EUCTR2010-022636-37-GR   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-GB   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-DK   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-BE   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-AT   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
   National Institute of Neurological Disorders and Stroke (NINDS)
      2004   Phase 4   NCT00097890   United States
   Sanofi
      2022   Phase 3   NCT05280548   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      2020   Phase 4   NCT04143958   Czechia
   Sanofi Aventis Groupe (SAG)
      2019   Phase 4   EUCTR2019-000064-21-GB   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
      -   Phase 4   EUCTR2019-000064-21-NO   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
      -   Phase 4   EUCTR2019-000064-21-CZ   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
   Shire
      2011   Phase 2   NCT01363492   United States
      2011   Phase 2   NCT01304277   Canada
      2011   Phase 3   NCT01298141   Canada
      2010   -   NCT01031173   United States
      2004   Phase 2   NCT00084084   Canada;United States
   Shire Human Genetic Therapies (HGT), Inc.
      2012   Phase 3;Phase 4   EUCTR2009-015985-75-SI   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
      2011   Phase 3;Phase 4   EUCTR2009-015985-75-GB   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
      2011   -   EUCTR2009-015985-75-FI   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
      2010   -   EUCTR2009-015985-75-PL   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
      2010   -   EUCTR2009-015985-75-CZ   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
   Shire Human Genetic Therapies, Inc.
      2022   Phase 3   EUCTR2018-004689-32-FI   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-SE   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PT   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PL   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-GR   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
   TKT Inc
      2004   -   EUCTR2004-000772-14-CZ   Czech Republic
   Takeda Development Center Americas, Inc
      -   Phase 3   EUCTR2022-004246-35-Outside-EU/EEA   China
   Tanaka Tomoyuki
      2022   Phase 3   JPRN-jRCT2071220022   China;Italy;Japan;Republic of Korea
   sanofi-aventis recherche et développement
      2022   Phase 3   EUCTR2021-002320-20-PL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NO   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-DK   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-CZ   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-AT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002320-20-GR   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-002320-20-ES   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
Agalsidase alpha
   Amicus Therapeutics
      2020   -   NCT04281537   Brazil;Japan;Taiwan;Turkey;United States
   Shire Human Genetic Therapies, Inc. (Shire HGT)
      2011   -   EUCTR2007-005543-22-SI   Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States
      2010   Phase 3;Phase 4   EUCTR2007-005543-22-GB   Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States
      2010   Phase 3;Phase 4   EUCTR2007-005543-22-FI   Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States
      2008   -   EUCTR2007-005543-22-CZ   Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States
   University Hospital, Rouen
      2017   -   NCT02956954   France
Agalsidase beta
   AMICUS THERAPEUTICS, INC
      2011   -   EUCTR2010-022636-37-IT   Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States
   Amicus Therapeutics
      2020   -   NCT04281537   Brazil;Japan;Taiwan;Turkey;United States
      2011   Phase 2   NCT01196871   Australia;Belgium;Canada;France;Netherlands;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2012   -   EUCTR2010-022709-16-GB   Belgium;United Kingdom
      2012   -   EUCTR2010-022636-37-DE   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   Phase 2   EUCTR2010-022709-16-BE   Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom
      2011   Phase 3   EUCTR2010-022636-37-GR   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-GB   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-DK   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-BE   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-AT   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
   Bio Sidus SA
      2022   Phase 3   NCT05843916   Argentina
   Genzyme Europe BV
      2010   -   EUCTR2006-001910-33-AT   Austria;United Kingdom
      2009   Phase 3   EUCTR2007-005668-28-FR   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-PT   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-NL   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-GB   Argentina;Brazil;Canada;Czech Republic;France;Germany;Netherlands;Norway;Poland;Portugal;United Kingdom;United States
      2008   -   EUCTR2007-005668-28-DE   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-CZ   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2007   -   EUCTR2006-001910-33-GB   Austria;United Kingdom
   Genzyme, a Sanofi Company
      2021   Phase 4   NCT05054387   China
      2012   Phase 4   NCT01650779   United States
      2008   Phase 3   NCT00701415   Argentina;Brazil;Canada;Chile;Czech Republic;Netherlands;Norway;Poland;Portugal;United Kingdom;United States
      2006   Phase 4   NCT00312767   United States
      2006   Phase 4   NCT00230607   Austria;United Kingdom;United States
      2005   Phase 4   NCT00140621   Japan
      2004   -   NCT00233870   Japan
      2004   Phase 4   NCT00081497   Canada;Czech Republic;Hungary;Poland;United Kingdom;United States
      2003   Phase 2   NCT00196716   Czech Republic;Estonia;Poland;Slovakia
      2002   Phase 2   NCT00837824   United States
      2002   Phase 2   NCT00074958   France;Italy;Poland;United Kingdom;United States
      2001   Phase 4   NCT00074984   Canada;Czech Republic;Hungary;Poland;United Kingdom;United States
      1999   Phase 3   NCT00074971   France;Netherlands;Puerto Rico;United Kingdom;United States
      -   -   EUCTR2015-000697-35-Outside-EU/EEA   United States
   ISU Abxis Co., Ltd.
      2023   Phase 3   NCT06081062   Korea, Republic of;Philippines
   JCR Pharmaceuticals Co., Ltd.
      2016   Phase 3   JPRN-jRCT2080223280   Japan
   Mackay Memorial Hospital
      2023   -   NCT05698901   Taiwan
   PROTALIX LTD
      2017   Phase 3   EUCTR2016-000378-38-IT   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
   Protalix
      2016   Phase 3   NCT02795676   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
   Protalix Ltd.
      2019   Phase 3   EUCTR2016-000378-38-FR   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2016-000378-38-FI   Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000378-38-SI   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000378-38-NL   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-NO   Australia;Belgium;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-HU   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-GB   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-ES   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-BE   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
   Sanofi
      2023   Phase 4   NCT06019728   United States
      2022   Phase 3   NCT05280548   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      2020   Phase 4   NCT04143958   Czechia
   Sanofi Aventis Groupe (SAG)
      2019   Phase 4   EUCTR2019-000064-21-GB   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
      -   Phase 4   EUCTR2019-000064-21-NO   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
      -   Phase 4   EUCTR2019-000064-21-CZ   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
      -   Phase 4   EUCTR2019-000065-20-NO   Canada;France;Norway;United States
   Tanaka Tomoyuki
      2022   Phase 3   JPRN-jRCT2071220022   China;Italy;Japan;Republic of Korea
   sanofi-aventis recherche et développement
      2022   Phase 3   EUCTR2021-002320-20-PL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NO   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-DK   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-CZ   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-AT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002320-20-GR   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-002320-20-ES   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
Agalsidase beta from biosidus 1 MG/KG
   Bio Sidus SA
      2021   Phase 1   NCT05343715   Argentina
Agalsidasi alfa
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
Agalsidasi beta
   FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
      2018   Phase 4   EUCTR2018-000368-27-IT   Italy
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
AGT-181
   ArmaGen, Inc
      2016   Phase 1/Phase 2   NCT03071341   Brazil
      2015   Phase 1/Phase 2   NCT03053089   Brazil
      2015   Phase 1   NCT02597114   -
      2015   Phase 1   NCT02371226   United States
AGT-182
   ArmaGen, Inc
      2015   Phase 1   NCT02262338   Germany;Netherlands;Philippines;United States
AL01211
   AceLink Therapeutics, Inc.
      2023   Phase 2   NCT06114329   China
Aldurazyme
      2007   -   EUCTR2007-001453-26-NL   Netherlands
   Genzyme Europe B. V
      -   Phase 4   EUCTR2023-001027-16-Outside-EU/EEA   China
   Genzyme, a Sanofi Company
      2005   Phase 3   NCT00258011   Japan
      2004   Phase 4   NCT00144781   Brazil;Canada
      2002   Phase 2   NCT00146757   France;Germany;Netherlands;United Kingdom
      2001   Phase 3   NCT00146770   Brazil;Canada;Germany;Italy;Netherlands;United Kingdom;United States
   HUS, Hospital for Children and Adolescents
      2007   -   EUCTR2006-005216-27-FI   Finland
   University of California, San Francisco
      2021   Phase 1   NCT04532047   United States
Aldurazyme*infus 1FL 5ML 500U
   Genzyme Europe BV
      2010   -   EUCTR2007-007003-33-IT   Italy
Alemtuzumab
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States
Alendronate sodium
   Children's Hospital Medical Center, Cincinnati
      1998   Phase 2   NCT00004488   Israel;United States
ALFA-manosidadas humana recombinante
   Zymenex A/S
      2012   -   EUCTR2011-004355-40-ES   Belgium;Denmark;Germany;Spain;United Kingdom
Allogeneic stem cell transplantation
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States
Ambroxol
   Exsar Corporation
      2012   Phase 1/Phase 2   NCT01463215   United States
   Maegaki Yoshihiro
      2019   Phase 3   JPRN-jRCTs061190017   -
      2010   Phase 2   JPRN-jRCTs061180090   -
   Shaare Zedek Medical Center
      2019   Phase 2   NCT03950050   Israel
   Tottori University, Faculty of Medicine, Institute of Neurological Science
      2012   Phase 2,3   JPRN-UMIN000009392   Japan
Ambroxol hydrochloride
   Amsterdam UMC
      2022   Phase 3   EUCTR2021-002550-82-NL   Netherlands
   Yutaka Shunichiro
      2021   Phase 1   JPRN-jRCT2051210024   -
Ambroxol hydrochloride 30 MG tablet - 18 MG/KG/DAY
   Ozlem Goker-Alpan
      2024   Phase 2/Phase 3   NCT06614894   United States
Ambroxol hydrochloride 30 MG tablet - 27 MG/KG/DAY
   Ozlem Goker-Alpan
      2024   Phase 2/Phase 3   NCT06614894   United States
Ambroxol hydrochloride 30 MG tablet - 9 MG/KG/DAY
   Ozlem Goker-Alpan
      2024   Phase 2/Phase 3   NCT06614894   United States
AMT-191
   UniQure Biopharma B.V.
      2024   Phase 1/Phase 2   NCT06270316   United States
Anakinra
   Lynda E Polgreen
      2020   Phase 1/Phase 2   NCT04018755   United States
Antihistamine
   Cinnagen
      2022   Phase 3   NCT06406153   Iran, Islamic Republic of
Antipyretic
   Cinnagen
      2022   Phase 3   NCT06406153   Iran, Islamic Republic of
Arimoclomol
   KemPharm Denmark A/S
      2018   Phase 2   NCT03746587   India
   Orphazyme ApS
      2016   Phase 2;Phase 3   EUCTR2015-004438-93-IT   Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-004438-93-ES   Denmark;Spain;Switzerland;United States
   ZevraDenmark
      2022   -   NCT04316637   United States
      2016   Phase 2/Phase 3   NCT02612129   Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States
Arimoclomol citrate
   Orphazyme A/S
      2016   Phase 2;Phase 3   EUCTR2015-004438-93-GB   Denmark;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-004438-93-DK   Denmark;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2015-004438-93-PL   Denmark;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States
   Zevra Denmark A/S
      2016   Phase 2;Phase 3   EUCTR2015-004438-93-DE   Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States
Arylsulfatase A
   Shire Pharmaceuticals Ireland Limited
      2006   -   EUCTR2006-005341-11-DK   Denmark
AT-GTX-501
   Amicus Therapeutics
      2016   Phase 1/Phase 2   NCT02725580   United States
   Amicus Therapeutics, Inc.
      2021   Phase 1;Phase 2   EUCTR2021-000706-21-DE   Germany;United States
AT1001
   AMICUS THERAPEUTICS, INC
      2011   -   EUCTR2010-022636-37-IT   Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2010   -   EUCTR2009-013459-31-IT   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
   Amicus Therapeutics
      2011   Phase 1   NCT01730482   -
      2005   Phase 2   EUCTR2005-004384-33-GB   United Kingdom
   Amicus Therapeutics UK, Ltd
      2020   Phase 3   EUCTR2017-000146-21-IT   Australia;Germany;Italy;Spain;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2015   Phase 3   EUCTR2014-002701-38-GB   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2015   Phase 3   EUCTR2014-002701-38-ES   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2015   Phase 3   EUCTR2014-002701-38-DK   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2015   Phase 3   EUCTR2014-002701-38-BE   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2014   Phase 3   EUCTR2014-002701-38-AT   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2014   Phase 3   EUCTR2011-004800-40-AT   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2013   Phase 3   EUCTR2011-004800-40-BE   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2012   Phase 3   EUCTR2011-004800-40-GB   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2012   Phase 3   EUCTR2011-004800-40-DK   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2012   -   EUCTR2010-022709-16-GB   Belgium;United Kingdom
      2012   -   EUCTR2010-022636-37-DE   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   Phase 2   EUCTR2010-022709-16-BE   Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom
      2011   Phase 3   EUCTR2010-022636-37-GR   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-GB   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-DK   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-BE   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-AT   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2010   Phase 3   EUCTR2009-013459-31-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2010   Phase 3   EUCTR2009-013459-31-ES   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2010   -   EUCTR2009-013459-31-DK   Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States
      2010   Phase 3   EUCTR2009-013459-31-BE   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 3   EUCTR2009-013459-31-GB   Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2009-013459-31-FR   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 2   EUCTR2007-001838-13-GB   Australia;Brazil;United Kingdom;United States
      2006   Phase 2   EUCTR2006-000181-36-GB   United Kingdom
      -   -   EUCTR2009-013459-31-DE   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
   Amicus Therapeutics, UK Limited
      2020   Phase 3   EUCTR2019-000222-21-GB   United Kingdom;United States
   Amicus Therapeutics, UK Ltd
      2019   Phase 3   EUCTR2017-000146-21-GB   Australia;Germany;Italy;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2017-000146-21-ES   Australia;Germany;Italy;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2017-000146-21-DE   Australia;Germany;Italy;Spain;United Kingdom;United States
   GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
      2012   -   EUCTR2011-004800-40-IT   Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States
   GlaxoSmithKline Research & Development Limited
      2012   Phase 3   EUCTR2011-004800-40-ES   Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
AT1001 150 MG
   Amicus Therapeutics
      2011   Phase 1   NCT01730469   United States
AT2101
   Amicus Therapeutics
      2009   Phase 1   NCT00875160   United Kingdom;United States
   Amicus Therapeutics, Inc.
      2009   -   EUCTR2008-007158-36-GB   United Kingdom
      2007   -   EUCTR2007-003359-35-GB   Germany;United Kingdom
      2007   -   EUCTR2007-003359-35-DE   Germany;United Kingdom
Ataluren
   PTC Therapeutics Inc.
      2015   Phase 2   EUCTR2015-003105-41-GB   United Kingdom
      2015   Phase 2   EUCTR2014-002596-28-GB   Germany;United Kingdom
ATG
   Alice Bertaina
      2023   Phase 1/Phase 2   NCT05508009   United States
Audiology assessment with ABR
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Autologous CD34+ cells transduced with A lentiviral vector containing THE human sgsh gene
   University of Manchester
      2020   Phase 1/Phase 2   NCT04201405   United Kingdom
      2019   Phase 1;Phase 2   EUCTR2019-002051-42-GB   United Kingdom
Autologous CD34+ cells transduced with A lentiviral vector encoding THE arsa cdna
   FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
      2010   Phase 1   EUCTR2009-017349-77-IT   Italy
Autologous CD34+ hscs transduced EX vivo with CD11B LV encoding human IDS tagged with apoeii
   University of Manchester
      2023   Phase 1/Phase 2   NCT05665166   United Kingdom
Autologous plasmablasts
   Immusoft of CA, Inc.
      2023   Phase 1   NCT05682144   United States
   Masonic Cancer Center, University of Minnesota
      2022   Phase 1/Phase 2   NCT04284254   -
AVR-RD-01
   AVROBIO
      2018   Phase 1/Phase 2   NCT03454893   Australia;Brazil;Canada;United States
AVR-RD-02
   AVROBIO
      2019   Phase 1/Phase 2   NCT04145037   Australia;Canada;United States
   John Bernat
      2024   -   NCT06488261   United States
AX 250
   Allievex Corporation
      2022   Phase 4   NCT05492799   Colombia;Germany;Turkey;United Kingdom;United States
      2022   Phase 3;Phase 4   EUCTR2022-002762-33-DE   Colombia;Germany;Turkey;United Kingdom;United States
      2018   Phase 2   NCT03784287   Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 2   EUCTR2017-003083-13-GB   Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 1/Phase 2   NCT02754076   Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-001985-25-DE   Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom
      2015   Phase 1;Phase 2   EUCTR2015-001985-25-GB   Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom
      -   Phase 2   EUCTR2017-003083-13-DE   Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States
AXO-AAV-GM2
   Terence Flotte
      2024   -   NCT06614569   United States
AXO-AAV-GM2 high dose
   Terence Flotte
      2021   Phase 1   NCT04669535   United States
AXO-AAV-GM2 LOW dose
   Terence Flotte
      2021   Phase 1   NCT04669535   United States
AXO-AAV-GM2 middle dose
   Terence Flotte
      2021   Phase 1   NCT04669535   United States
AXO-AAV-GM2 starting dose
   Terence Flotte
      2021   Phase 1   NCT04669535   United States
AZ-3102
   Azafaros A.G.
      2023   Phase 2   NCT05758922   Brazil;United States
Azathioprine
   Genzyme, a Sanofi Company
      2008   Phase 1/Phase 2   NCT00741338   Brazil;Russian Federation;Ukraine
BBM-F101 injection
   Children's Hospital of Fudan University
      2024   Early Phase 1   NCT06207552   China
Biological assays
   University Hospital, Bordeaux
      2016   -   NCT03123523   France
Blood draw
   Baylor Research Institute
      2018   -   NCT04002531   United States
Blood sampling FOR biological and genetic analysis
   University Hospital, Bordeaux
      2010   -   NCT01178164   France
BM110
   BIOMARIN PHARMACEUTICAL INC.
      2011   -   EUCTR2010-020199-45-IT   Argentina;Australia;Brazil;Canada;Colombia;Denmark;Germany;Italy;Japan;Korea, Democratic People's Republic of;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States
BMN 110
   BioMarin Pharmaceutical
      2013   Phase 3   NCT01966029   Australia
      2012   Phase 2   NCT01697319   Germany;United Kingdom;United States
      2012   Phase 2   NCT01609062   Canada;Germany;United Kingdom;United States
      2011   Phase 2   NCT01515956   Italy;Taiwan;United Kingdom;United States
      2010   Phase 1/Phase 2   NCT01242111   United Kingdom
      2009   -   NCT01858103   Puerto Rico;United States
      2009   Phase 1/Phase 2   NCT00884949   United Kingdom
   BioMarin Pharmaceutical Inc.
      2009   -   EUCTR2008-007365-23-GB   United Kingdom
BMN 110 - every other week
   BioMarin Pharmaceutical
      2011   Phase 3   NCT01415427   Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States
BMN 110 - weekly
   BioMarin Pharmaceutical
      2011   Phase 3   NCT01415427   Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States
BMN 110 every other week
   BioMarin Pharmaceutical
      2011   Phase 3   NCT01275066   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;Turkey;United Kingdom;United States
BMN 110 weekly
   BioMarin Pharmaceutical
      2011   Phase 3   NCT01275066   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;Turkey;United Kingdom;United States
BMN 190
   BioMarin Pharmaceutical
      2015   Phase 1/Phase 2   NCT02485899   Germany;Italy;United Kingdom;United States
      2013   Phase 1/Phase 2   NCT01907087   Germany;Italy;United Kingdom;United States
   BioMarin Pharmaceutical Inc
      2017   Phase 2   EUCTR2015-000891-85-GB   Germany;Italy;United Kingdom;United States
   BioMarin Pharmaceutical Inc.
      2016   Phase 2   EUCTR2015-000891-85-DE   Germany;Italy;United Kingdom
      2015   -   EUCTR2014-003480-37-IT   Germany;Italy;Japan;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003480-37-GB   Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003480-37-DE   Germany;Italy;United Kingdom;United States
      2014   Phase 1;Phase 2   EUCTR2012-005430-11-IT   Germany;Italy;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2012-005430-11-GB   Germany;Italy;United Kingdom;United States
      2013   Phase 1;Phase 2   EUCTR2012-005430-11-DE   Germany;United Kingdom
BMN 250
   BioMarin Pharmaceutical Inc.
      2017   Phase 2   EUCTR2017-003083-13-ES   Argentina;Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-001985-25-ES   Australia;Brazil;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom
Brain MRI/MRS/fmri
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Brief pain inventory questionnaire
   Baylor Research Institute
      2018   -   NCT04002531   United States
Brineura
   BioMarin Pharmaceutical Inc
      2017   Phase 2   EUCTR2015-000891-85-GB   Germany;Italy;United Kingdom;United States
   BioMarin Pharmaceutical Inc.
      2016   Phase 2   EUCTR2015-000891-85-DE   Germany;Italy;United Kingdom
      2015   Phase 2   EUCTR2014-003480-37-GB   Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003480-37-DE   Germany;Italy;United Kingdom;United States
Busilvex
   FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
      2010   Phase 1   EUCTR2009-017349-77-IT   Italy
Busilvex - 6 MG/ML - concentrato PER soluzione PER infusione - USO endovenoso - flaconcino - 10 ML 8 flaconcini
   OSPEDALE SAN RAFFAELE
      2018   Phase 1   EUCTR2017-002430-23-IT   Italy
Busulfan
   FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
      2010   Phase 1   EUCTR2009-017349-77-IT   Italy
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States
      2008   Phase 2   NCT00668564   United States
Busulfan, cyclophosphamide, antithymocyte globulin
   Masonic Cancer Center, University of Minnesota
      1995   Phase 2/Phase 3   NCT00176904   United States
Busulfan, cyclophosphamide, ATG
   Masonic Cancer Center, University of Minnesota
      1999   Phase 2   NCT00176917   United States
Busulfano
   OSPEDALE SAN RAFFAELE
      2018   Phase 1   EUCTR2017-002430-23-IT   Italy
Calcium carbonate
   Children's Hospital Medical Center, Cincinnati
      1998   Phase 2   NCT00004488   Israel;United States
Campath-1H
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
      2009   Phase 2   NCT01043640   United States
      2008   Phase 2   NCT00668564   United States
Cannabidiol
   Amsterdam UMC
      2025   Phase 3   EUCTR2021-003250-23-NL   Netherlands
Cannabis sativa L., folium CUM flore
   Albina Nowak, MD
      2022   -   NCT04820361   Switzerland
Cebsulfase alfa
   Shire Human Genetics Therapies Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited
      2013   Phase 1;Phase 2   EUCTR2012-003775-20-DK   Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2012-003775-20-DE   Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom
Cellule CD34+ autologhe trasdotte CON idua LV codificante PER IL cdna dell'ALFA-L-iduronidasi
   OSPEDALE SAN RAFFAELE
      2018   Phase 1   EUCTR2017-002430-23-IT   Italy
Cerdelga
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000301-37-NL   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2019   Phase 3   EUCTR2016-000301-37-GB   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-SE   Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-IT   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-ES   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
Cerezyme
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000301-37-NL   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2019   Phase 3   EUCTR2016-000301-37-GB   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-SE   Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-IT   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-ES   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
   Genzyme Europe B.V.
      2010   -   EUCTR2008-005223-28-DE   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2008-005223-28-NL   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005223-28-GB   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2008-005223-28-FR   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   -   EUCTR2008-005223-28-CZ   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
   Genzyme Europe BV
      2010   -   EUCTR2008-005223-28-IT   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
   Genzyme, a Sanofi Company
      2001   Phase 4   NCT00364858   Brazil;Canada;Italy;Poland;Spain;United Kingdom;United States
      1997   Phase 4   NCT00365131   United States
   INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD
      2008   -   EUCTR2007-007164-20-ES   Spain
   ISTITUTO GIANNINA GASLINI
      2009   -   EUCTR2007-005516-61-IT   Italy
   ISU Abxis Co., Ltd.
      2011   Phase 3   NCT01161914   -
   Sanofi-aventis recherche & développement
      -   Phase 3   EUCTR2021-005402-10-FR   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
   Shire Human Genetic Therapies Inc
      2007   Phase 3   EUCTR2007-002840-21-ES   Italy;Spain;United Kingdom
   Shire Human Genetic Therapies, Inc.
      2007   -   EUCTR2007-002840-21-GB   Italy;Spain;United Kingdom
   Tanaka Tomoyuki
      2023   Phase 3   JPRN-jRCT2021220029   Argentina;Canada;China;France;Germany;Hungary;Japan;United States
Cerezyme 400 units powder FOR concentrate FOR solution FOR infusion
   Sanofi-aventis recherche & développement
      2022   Phase 3   EUCTR2021-005402-10-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005402-10-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Türkiye;United Kingdom;United States
Cerezyme 400 unità polvere PER concentrato PER soluzione PER infusione
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2023   Phase 3   EUCTR2021-005402-10-IT   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
Cerezyme® / imiglucerase
   Sanofi
      2021   Phase 4   NCT04656600   China
Cerliponase alfa
   BioMarin Pharmaceutical
      2020   -   NCT04476862   United States
   BioMarin Pharmaceutical Inc
      2017   Phase 2   EUCTR2015-000891-85-GB   Germany;Italy;United Kingdom;United States
   BioMarin Pharmaceutical Inc.
      2016   Phase 2   EUCTR2015-000891-85-DE   Germany;Italy;United Kingdom
      2015   Phase 2   EUCTR2014-003480-37-GB   Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003480-37-DE   Germany;Italy;United Kingdom;United States
      2013   Phase 1;Phase 2   EUCTR2012-005430-11-GB   Germany;Italy;United Kingdom;United States
   David L Rogers, MD
      2021   Phase 1/Phase 2   NCT05152914   United States
Cetirizine
   Sanofi
      2023   Phase 4   NCT06019728   United States
Challenge agent: capsaicin
   Universitaire Ziekenhuizen Leuven
      2014   Phase 0   NCT02533076   Belgium
Chemically modified recombinant human sulfamidase
   Swedish Orphan Biovitrum AB (publ)
      2019   Phase 1   EUCTR2017-002806-10-NL   Germany;Netherlands;Turkey;United States
      -   Phase 1   EUCTR2017-002806-10-DE   Germany;Netherlands;Turkey;United States
CHF6657
   Chiesi Farmaceutici S.p.A
      2021   Phase 3   EUCTR2018-001148-67-FI   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2018-001148-67-SI   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001947-30-DK   Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001148-67-NL   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001148-67-HU   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001148-67-NO   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001148-67-CZ   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
   Chiesi Farmaceutici S.p.A.
      2019   Phase 3   EUCTR2018-001947-30-BE   Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States
Cholecalciferol
   Children's Hospital Medical Center, Cincinnati
      1998   Phase 2   NCT00004488   Israel;United States
Cholesterol
   Alexion Pharmaceuitcals Inc
      2014   Phase 2   EUCTR2011-004287-30-HR   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-DE   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals Inc
      2015   Phase 2   EUCTR2014-000533-22-FI   Finland;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2011-004287-30-NL   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2015   Phase 2   EUCTR2011-004287-30-GB   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2014-000533-22-GB   Finland;Italy;United Kingdom;United States
   Alexion Pharmaceuticals inc
      2014   Phase 2   EUCTR2011-004287-30-DK   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals, Inc.
      2015   Phase 3   EUCTR2011-002750-31-CZ   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-GB   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-DE   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      -   Phase 3   EUCTR2011-002750-31-PL   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
   Synageva BioPharma Corp.
      2014   Phase 2   EUCTR2014-000533-22-IT   Finland;Italy;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-IT   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-BE   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
   Synageva BioPharma Corporation
      2013   Phase 3   EUCTR2011-002750-31-IT   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-HR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-GR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-ES   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      -   Phase 3   EUCTR2011-002750-31-FR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
   Synageva Biopharma Corp.
      2011   Phase 2   EUCTR2011-001513-13-GB   Czech Republic;France;United Kingdom;United States
      2011   Phase 2   EUCTR2011-001513-13-CZ   Czech Republic;France;United Kingdom;United States
Clinimacs® TCR A/ß reagent KIT and clinimacs® CD19 system
   Alice Bertaina
      2023   Phase 1/Phase 2   NCT05508009   United States
Clofarabine
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
      2006   Phase 2   NCT00383448   United States
Contrast sensitivity measurement
   Université de Montréal
      2014   -   NCT02023086   Canada
Coversyl plus
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Cryopreserved autologous CD34+ cells transduced with A lentiviral vector containing human sgsh gene
   University of Manchester
      2019   Phase 1;Phase 2   EUCTR2019-002051-42-GB   United Kingdom
CSA
   Genzyme, a Sanofi Company
      2008   Phase 1/Phase 2   NCT00741338   Brazil;Russian Federation;Ukraine
Cyclophosphamide
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States
      2008   Phase 2   NCT00668564   United States
Cyclophosphamide 100 MG/KG
   Alice Bertaina
      2023   Phase 1/Phase 2   NCT05508009   United States
Cyclophosphamide 1200 MG/KG
   Alice Bertaina
      2023   Phase 1/Phase 2   NCT05508009   United States
Cyclosporine A
   Genzyme, a Sanofi Company
      2008   Phase 1/Phase 2   NCT00741338   Brazil;Russian Federation;Ukraine
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
      2009   Phase 2   NCT01043640   United States
      2006   Phase 2   NCT00383448   United States
Cystadane anhydrous
   Minna Laine
      2017   Phase 2   EUCTR2017-000645-48-FI   Finland
Cystadrops
   Orphan Europe SARL
      2008   -   EUCTR2007-006024-35-FR   France
   RECORDATI Rare Diseases SARL
      2019   Phase 3   EUCTR2018-002984-24-GB   France;United Kingdom
   Recordati Rare Diseases
      2021   Phase 3   EUCTR2018-002984-24-BE   Belgium;France;Germany;Italy;United Kingdom
      2020   Phase 3   EUCTR2018-002984-24-IT   Belgium;France;Germany;Italy;United Kingdom
      2020   Phase 3   EUCTR2018-002984-24-DE   France;Germany;United Kingdom
      -   Phase 3   EUCTR2018-002984-24-FR   France;United Kingdom
Cystadrops 0.55% EYE drops, solution
   Orphan Europe SARL
      2013   -   EUCTR2009-012564-13-FR   France
Cystagon
   Amgen
      2013   Phase 3   NCT01733316   Belgium;France;Italy;Netherlands;United Kingdom;United States
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      2001   Phase 4   NCT00028262   United States
   HOSPICES CIVILS DE LYON
      2010   -   EUCTR2010-020098-18-FR   France
   Horizon Pharma USA, Inc.
      2010   Phase 3   NCT01000961   France;Netherlands;United States
      2009   Phase 1/Phase 2   NCT00872729   United States
   Raptor Therapeutics Inc.
      2014   Phase 3   EUCTR2012-002773-64-IT   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2013   Phase 3   EUCTR2012-002773-64-FR   Belgium;France;Italy;Netherlands;United Kingdom;United States
      2010   -   EUCTR2009-017882-42-NL   France;Netherlands
      2010   Phase 3   EUCTR2009-017882-42-FR   France;Netherlands
Cystagon 150MG
   Horizon Pharma USA, Inc.
      2014   Phase 3   EUCTR2012-002773-64-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2013   Phase 3   EUCTR2012-002773-64-NL   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
   Raptor Therapeutics Inc.
      2014   Phase 3   EUCTR2012-002773-64-BE   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
Cystagon 50MG
   Horizon Pharma USA, Inc.
      2014   Phase 3   EUCTR2012-002773-64-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2013   Phase 3   EUCTR2012-002773-64-NL   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
   Raptor Therapeutics Inc.
      2014   Phase 3   EUCTR2012-002773-64-BE   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
Cysteamine
   HOSPICES CIVILS DE LYON
      2010   -   EUCTR2010-020098-18-FR   France
   National Eye Institute (NEI)
      1998   Phase 1   NCT00001736   United States
      1986   Phase 2   NCT00001213   United States
   National Human Genome Research Institute (NHGRI)
      1979   -   NCT00359684   United States
   Yamamoto Yuuka
      2022   Phase 3   JPRN-jRCTs031220490   -
Cysteamine (mercaptamine) viscous solution
   Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR)
      2014   -   EUCTR2013-003228-35-ES   Spain
Cysteamine bitartrate
   Chiesi SA/NV
      2020   -   NCT04246060   Belgium
   Hospices Civils de Lyon
      2011   -   NCT02012114   France
   Nacuity Pharmaceuticals, Inc.
      2023   Phase 1/Phase 2   NCT05994534   Australia
   University of California, San Diego
      2011   -   NCT01432561   United States
Cysteamine bitartrate (INN: mercaptamine bitartrate
   Horizon Pharma USA, Inc.
      2014   Phase 3   EUCTR2012-002773-64-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2013   Phase 3   EUCTR2012-002773-64-NL   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2010   Phase 3   EUCTR2010-018365-34-NL   France;Netherlands;United States
   Raptor Therapeutics Inc.
      2014   Phase 3   EUCTR2012-002773-64-IT   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 3   EUCTR2012-002773-64-BE   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2013   Phase 3   EUCTR2012-002773-64-FR   Belgium;France;Italy;Netherlands;United Kingdom;United States
      2010   -   EUCTR2009-017882-42-NL   France;Netherlands
      2010   Phase 3   EUCTR2009-017882-42-FR   France;Netherlands
      -   Phase 3   EUCTR2010-018365-34-FR   France;Netherlands
Cysteamine bitartrate delayed-release capsules
   Amgen
      2010   Phase 3   NCT01197378   France;Netherlands;United States
   Horizon Pharma USA, Inc.
      2010   Phase 3   NCT01000961   France;Netherlands;United States
Cysteamine hydrochloride
   FDA Office of Orphan Products Development
      1999   -   NCT00010426   -
   Orphan Europe SARL
      2013   -   EUCTR2009-012564-13-FR   France
      2008   -   EUCTR2007-006024-35-FR   France
   RECORDATI Rare Diseases SARL
      2019   Phase 3   EUCTR2018-002984-24-GB   France;United Kingdom
   Recordati Rare Diseases
      2021   Phase 3   EUCTR2018-002984-24-BE   Belgium;France;Germany;Italy;United Kingdom
      2020   Phase 3   EUCTR2018-002984-24-DE   France;Germany;United Kingdom
Dapagliflozin 10MG TAB
   Albina Nowak, MD
      2023   Phase 2   NCT05710367   -
Deflegmin 75MG, kapsulki O przedluzonym uwalnianiu
   Instytut ,,Pomnik-Centrum Zdrowia Dziecka
      -   Phase 2   EUCTR2021-006224-40-PL   Poland
Dexa scan
   University of Texas Southwestern Medical Center
      2006   -   NCT00286689   United States
Dexamethasone
   Sanofi
      2023   Phase 4   NCT06019728   United States
DFT383
   Novartis Pharmaceuticals
      2025   Phase 1/Phase 2   NCT06910813   United States
Diagnosis OF fabry disease
   University Hospital, Bordeaux
      2015   -   NCT02450604   France
Difenidramina cloridrato
   Sangamo Therapeutics, Inc.
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-IT   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
Diphenhydramine
   Sanofi
      2023   Phase 4   NCT06019728   United States
DNL126
   Denali Therapeutics Inc.
      2023   Phase 1/Phase 2   NCT06181136   United States
DNL310 drug substance
   Denali Therapeutics Inc.
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-SE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-NL   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-IT   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-FR   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-CZ   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-BE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2021   Phase 1;Phase 2   EUCTR2019-004909-27-NL   Germany;Italy;Netherlands;United Kingdom;United States
      2020   Phase 1;Phase 2   EUCTR2019-004909-27-GB   Germany;Italy;Netherlands;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-005200-35-DE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
Dried blood spot (DBS) sampling
   Hospices Civils de Lyon
      2016   -   NCT02843334   France
DRX005B
   National Institute of Neurological Disorders and Stroke (NINDS)
      2002   Phase 2   NCT00048906   United States
   Shire Human Genetic Therapies, Inc.
      2022   Phase 3   EUCTR2018-004689-32-FI   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-SE   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PT   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PL   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-GR   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
DUOC-01
   Joanne Kurtzberg, MD
      2014   Phase 1   NCT02254863   United States
Echocardiogram
   Baylor Research Institute
      2018   -   NCT04002531   United States
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Echocardiography AT M24
   University Hospital, Bordeaux
      2016   -   NCT03123523   France
Echocardiography AT T0
   University Hospital, Bordeaux
      2016   -   NCT03123523   France
Elaprase
      2007   -   EUCTR2007-001453-26-NL   Netherlands
   Denali Therapeutics Inc.
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-SE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-NL   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-IT   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-FR   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-CZ   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-BE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-005200-35-DE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
   JCR Pharmaceutical Co., Ltd.
      2023   Phase 3   EUCTR2020-003200-14-IT   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Turkey;United Kingdom;United States
   JCR Pharmaceuticals Co., Ltd.
      2023   Phase 3   EUCTR2020-003200-14-PL   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003200-14-DE   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Türkiye;United Kingdom;United States
   Shire
      2015   Phase 2/Phase 3   NCT02412787   Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States
      2010   Phase 1/Phase 2   NCT01506141   Canada;United Kingdom;United States
   Shire Human Genetic Therapies, Inc. (Shire), a wholly owned subsidiary of Takeda Pharmaceutical Company Limited
      2016   Phase 4   EUCTR2014-004804-31-DE   Costa Rica;Dominican Republic;Germany;Malaysia;Oman;Philippines;Saudi Arabia;Serbia;Thailand;United States;Viet Nam
   Takeda
      2024   Phase 2/Phase 3   NCT06031259   Canada;France
      2023   Phase 4   NCT05494593   United States
      2022   Phase 4   NCT05058391   India
   Takeda Biopharmaceuticals India Pvt. Ltd.
      -   Phase 4   EUCTR2022-004193-39-Outside-EU/EEA   India
Elaprase 2MG/ML
   Shire Human Genetic Therapies, Inc. (Shire HGT)
      -   Phase 4   EUCTR2007-006044-22-PL   Poland
Elaprase FOR intravenous (IV) infusion
   Shire
      2015   Phase 4   NCT02455622   Dominican Republic;Germany;Malaysia;Oman;Philippines;Saudi Arabia;Serbia;Thailand;United States;Vietnam
Elaprase_0.5MG/KG
   Green Cross Corporation
      2010   Phase 1/Phase 2   NCT01301898   Korea, Republic of
Electroencephalogram (EEG) awake and extended overnight
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Elelyso
   Ari Zimran
      2020   Phase 4   NCT04002830   India;Israel;Turkey
   Shaare Zedek Medical Center
      2017   -   NCT04353466   -
Elelyso 60 units/KG
   Pfizer
      2019   Phase 4   NCT03021941   United States
Elfabrio
   Chiesi Farmaceutici S.p.A
      2019   Phase 3   EUCTR2018-001947-30-DK   Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States
Eliglustat
   Genzyme Europe B.V.
      2011   Phase 3   EUCTR2009-015811-42-PT   Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States
      2011   -   EUCTR2009-015811-42-GR   Austria;Greece;Portugal;Sweden
      2011   Phase 3   EUCTR2009-015811-42-AT   Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States
      2010   Phase 3   EUCTR2009-015811-42-SE   Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States
      2010   -   EUCTR2008-005223-28-DE   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2010   -   EUCTR2008-005222-37-BG   Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States
      2009   Phase 3   EUCTR2008-005223-28-NL   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005223-28-GB   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2009   -   EUCTR2008-005222-37-NL   Bulgaria;Netherlands;United Kingdom
      2009   Phase 3   EUCTR2008-005222-37-GB   Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States
   Genzyme, a Sanofi Company
      2015   Phase 1   NCT02536911   United States
      2015   Phase 1   NCT02422654   United States
   National Taiwan University Hospital
      2018   -   NCT03519646   Taiwan
   Sanofi
      2018   Phase 1   NCT06188325   United States
      2015   Phase 1   NCT02536937   United States
      2014   Phase 1   NCT06193304   United States
Eliglustat GZ385660
   Sanofi
      2018   Phase 3   NCT03485677   Argentina;Canada;France;Italy;Japan;Russian Federation;Spain;Sweden;Turkey;United Kingdom
Eliglustat tartrate
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000301-37-NL   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2019   Phase 3   EUCTR2016-000301-37-GB   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-IT   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-ES   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
   Genzyme Europe B.V.
      2009   -   EUCTR2008-005223-28-CZ   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
   Genzyme, a Sanofi Company
      2010   Phase 3   NCT01074944   Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States
      2009   Phase 3   NCT00943111   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   NCT00891202   Bulgaria;Canada;Chile;Colombia;Georgia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States
      2006   Phase 2   NCT00358150   Argentina;Israel;Italy;Mexico;Russian Federation;United States
Eliglustat tartrate capsules
   Peking Union Medical College Hospital
      2024   Phase 2   NCT06523517   China
Eliglustat tartrate – powder FOR oral suspension
   Genzyme Corporation
      2018   Phase 3   EUCTR2016-000301-37-SE   Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom
Elosulfase alfa
   BioMarin Pharmaceutical
      2014   -   NCT02294877   Australia;Austria;Belgium;Canada;Czechia;Denmark;France;Germany;Ireland;Italy;Malaysia;Netherlands;Poland;Portugal;Puerto Rico;Taiwan;United Kingdom;United States
   GOIZET
      2018   -   NCT03204370   France
ELX-02
   Eloxx Pharmaceuticals, Inc.
      2019   Phase 2   NCT04069260   Canada
Enalapril and other angiotensin converting enzyme inhibitors
   University of Alabama at Birmingham
      2007   -   NCT00446862   Germany;Slovenia;United States
Enriched hematopoetic stem cell infusion
   Talaris Therapeutics Inc.
      2013   -   NCT02021266   United States
Enzyme replacement
   Rigshospitalet, Denmark
      2001   Phase 4   NCT01997489   Denmark
Enzyme replacement agent
   AVROBIO
      2023   Phase 2/Phase 3   NCT05815004   -
Enzyme replacement therapy
   Idorsia Pharmaceuticals Ltd.
      2015   Phase 1   NCT02930655   Germany
   Ulla Feldt-Rasmussen
      2015   -   NCT02969200   Denmark
   University Hospital, Rouen
      2017   -   NCT02956954   France
Epidiolex
   Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
      2025   Phase 2/Phase 3   NCT06333041   United States
ERT
   Amicus Therapeutics
      2025   -   NCT06906367   -
   Idorsia Pharmaceuticals Ltd.
      2015   Phase 1   NCT02930655   Germany
   National Taiwan University Hospital
      2015   -   NCT02520934   Taiwan
EU-sourced cerezyme
   ISU Abxis Co., Ltd.
      2020   Phase 1   NCT04787887   Australia
Exercise test
   University Hospital, Bordeaux
      2016   -   NCT03123523   France
EXG110 injection
   Hangzhou Jiayin Biotech Ltd
      2025   Phase 1/Phase 2   NCT06819514   China
   The Children's Hospital of Zhejiang University School of Medicine
      2024   -   NCT06539624   China
Fabrazyme
   AMICUS THERAPEUTICS, INC
      2011   -   EUCTR2010-022636-37-IT   Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2012   -   EUCTR2010-022709-16-GB   Belgium;United Kingdom
      2012   -   EUCTR2010-022636-37-DE   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   Phase 2   EUCTR2010-022709-16-BE   Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom
      2011   Phase 3   EUCTR2010-022636-37-GR   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-GB   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-DK   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-BE   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-AT   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
   Genzyme Europe BV
      2010   -   EUCTR2006-001910-33-AT   Austria;United Kingdom
      2009   Phase 3   EUCTR2007-005668-28-FR   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-PT   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-NL   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-GB   Argentina;Brazil;Canada;Czech Republic;France;Germany;Netherlands;Norway;Poland;Portugal;United Kingdom;United States
      2008   -   EUCTR2007-005668-28-CZ   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2007   -   EUCTR2006-001910-33-GB   Austria;United Kingdom
   Genzyme, a Sanofi Company
      2006   Phase 4   NCT00312767   United States
      2003   Phase 2   NCT00196716   Czech Republic;Estonia;Poland;Slovakia
      2002   Phase 2   NCT00837824   United States
      2002   Phase 2   NCT00074958   France;Italy;Poland;United Kingdom;United States
      2001   Phase 4   NCT00074984   Canada;Czech Republic;Hungary;Poland;United Kingdom;United States
      1999   Phase 3   NCT00074971   France;Netherlands;Puerto Rico;United Kingdom;United States
   Protalix Ltd.
      2019   Phase 3   EUCTR2016-000378-38-FR   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2016-000378-38-FI   Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000378-38-SI   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000378-38-NL   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-NO   Australia;Belgium;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-HU   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-GB   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-ES   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-BE   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
   Sanofi Aventis Groupe (SAG)
      2019   Phase 4   EUCTR2019-000064-21-GB   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
      -   Phase 4   EUCTR2019-000065-20-NO   Canada;France;Norway;United States
      -   Phase 4   EUCTR2019-000064-21-NO   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
      -   Phase 4   EUCTR2019-000064-21-CZ   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
   sanofi-aventis recherche et développement
      2022   Phase 3   EUCTR2021-002320-20-PL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NO   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-DK   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-CZ   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-AT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002320-20-GR   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-002320-20-ES   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
Fabrazyme (agalsidase beta) 1 MG/KG
   Bio Sidus SA
      2021   Phase 1   NCT05343715   Argentina
Fabrazyme - 35 MG polvere PER concentrato PER soluzione PER infusione endovenosa 1 flaconcino
   FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
      2018   Phase 4   EUCTR2018-000368-27-IT   Italy
   PROTALIX LTD
      2017   Phase 3   EUCTR2016-000378-38-IT   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
Fabrazyme 35 MG
   Genzyme Europe BV
      2008   -   EUCTR2007-005668-28-DE   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
Fabrazyme 5 MG
   Genzyme Europe BV
      2008   -   EUCTR2007-005668-28-DE   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
FBX-101
   Forge Biologics, Inc
      2024   -   NCT06308718   United States
      2023   Phase 1/Phase 2   NCT05739643   United States
      2021   Phase 1/Phase 2   NCT04693598   United States
Fexofenadine
   Sanofi
      2023   Phase 4   NCT06019728   United States
FLT190
   Freeline Therapeutics
      2020   Phase 1/Phase 2   NCT04455230   Germany;United Kingdom
      2019   Phase 1/Phase 2   NCT04040049   Austria;Canada;Germany;Italy;Norway;United Kingdom;United States
   Freeline Therapeutics Limited
      2019   Phase 1;Phase 2   EUCTR2018-002097-51-IT   Austria;Denmark;France;Germany;Italy;Norway;United Kingdom;United States
   Freeline Therapeutics Ltd
      2022   Phase 2   EUCTR2019-004645-32-DE   Austria;Canada;Denmark;France;Germany;Italy;Norway;Portugal;United Kingdom;United States
      2021   Phase 1;Phase 2   EUCTR2018-002097-51-AT   Austria;Denmark;France;Germany;Italy;Norway;Switzerland;United Kingdom;United States
      2020   Phase 2   EUCTR2019-004645-32-GB   Denmark;France;Germany;Italy;Norway;United Kingdom;United States
      2020   Phase 1;Phase 2   EUCTR2018-002097-51-DK   Denmark;France;Germany;Italy;Norway;Spain;United Kingdom;United States
      2020   Phase 1;Phase 2   EUCTR2018-002097-51-DE   Austria;Denmark;France;Germany;Italy;Norway;Spain;United Kingdom;United States
FLT201
   Freeline Therapeutics
      2024   Phase 1/Phase 2   NCT06545136   Brazil;Spain;United Kingdom;United States
   Spur Therapeutics
      2022   Phase 1   NCT05324943   Argentina;Brazil;Germany;Israel;Italy;Paraguay;Spain;Tunisia;United Kingdom;United States
Fludarabina accord - 25 MG/ML concentrato PER soluzione iniettabile O PER infusione 5 flaconcini IN vetro DA 2 ML
   OSPEDALE SAN RAFFAELE
      2018   Phase 1   EUCTR2017-002430-23-IT   Italy
Fludarabina fosfato
   OSPEDALE SAN RAFFAELE
      2018   Phase 1   EUCTR2017-002430-23-IT   Italy
Fludarabine
   Alice Bertaina
      2023   Phase 1/Phase 2   NCT05508009   United States
Fluorescein angiography
   Ohio State University
      2010   -   NCT01165697   United States
GA-GCB
   SHIRE HUMAN GENETIC THERAPIES, INC
      2018   Phase 4   EUCTR2015-001578-17-IT   Germany;Israel;Italy;Spain;United Kingdom;United States
   SHIRE HUMAN GENETIC THERAPIES, INC.
      2008   -   EUCTR2007-002840-21-IT   Italy;Spain;United Kingdom
      2008   -   EUCTR2006-006304-11-IT   Italy;Spain;United Kingdom
   Shire
      2007   Phase 2/Phase 3   NCT00478647   Israel;Poland;Spain;United Kingdom;United States
      2005   Phase 1/Phase 2   NCT00391625   Former Serbia and Montenegro;Israel;Romania;Serbia
   Shire Human Genetic Therapies
      -   -   EUCTR2012-003427-38-Outside-EU/EEA   Egypt;India;Tunisia
   Shire Human Genetic Therapies Inc
      2007   Phase 3   EUCTR2007-002840-21-ES   Italy;Spain;United Kingdom
      2007   Phase 2;Phase 3   EUCTR2006-006304-11-GB   Germany;Italy;Spain;United Kingdom
      2007   Phase 2;Phase 3   EUCTR2006-006304-11-ES   Germany;Italy;Spain;United Kingdom
      -   Phase 2;Phase 3   EUCTR2006-006304-11-DE   Germany;Italy;Spain;United Kingdom
   Shire Human Genetic Therapies, Inc.
      2016   Phase 4   EUCTR2015-001578-17-GB   Canada;Germany;Israel;Spain;United Kingdom;United States
      2016   Phase 4   EUCTR2015-001578-17-ES   Canada;Germany;Israel;Italy;Spain;United Kingdom;United States
      2016   Phase 4   EUCTR2015-001578-17-DE   Germany;India;Israel;Italy;Spain;Turkey;United Kingdom;United States
      2008   Phase 3   EUCTR2008-001965-27-GB   Spain;United Kingdom
      2008   -   EUCTR2008-001965-27-ES   Spain;United Kingdom
      2007   -   EUCTR2007-002840-21-GB   Italy;Spain;United Kingdom
Gabapentin
   University of Minnesota
      2012   Phase 2   NCT01588314   United States
Galafold
   Amicus Therapeutics UK, Ltd
      2020   Phase 3   EUCTR2017-000146-21-IT   Australia;Germany;Italy;Spain;United Kingdom;United States
   Amicus Therapeutics, UK Ltd
      2019   Phase 3   EUCTR2017-000146-21-GB   Australia;Germany;Italy;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2017-000146-21-ES   Australia;Germany;Italy;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2017-000146-21-DE   Australia;Germany;Italy;Spain;United Kingdom;United States
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
   Sanofi Aventis Groupe (SAG)
      -   Phase 4   EUCTR2019-000065-20-NO   Canada;France;Norway;United States
   sanofi-aventis recherche et développement
      2022   Phase 3   EUCTR2021-002320-20-PL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NO   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-DK   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-CZ   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-AT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002320-20-GR   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-002320-20-ES   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
Galsulfase
      2007   -   EUCTR2007-001453-26-NL   Netherlands
   BioMarin Pharmaceutical Inc
      2007   -   EUCTR2005-003512-30-PT   France;Portugal
   BioMarin Pharmaceutical Inc.
      2007   Phase 4   EUCTR2005-003512-30-FR   France;Portugal
GC1111_0.5MG/KG
   Green Cross Corporation
      2010   Phase 1/Phase 2   NCT01301898   Korea, Republic of
GC1111_1.0MG/KG
   Green Cross Corporation
      2010   Phase 1/Phase 2   NCT01301898   Korea, Republic of
GC1119
   Green Cross Corporation
      2012   Phase 1   NCT01653444   Korea, Republic of
GC1123
   GC Biopharma Corp
      2022   Phase 1   NCT05422482   Korea, Republic of
GC1130A
   GC Biopharma Corp
      2024   Phase 1   NCT06567769   Japan;Korea, Republic of;United States
Gelatine
   Zevra Denmark A/S
      2016   Phase 2;Phase 3   EUCTR2015-004438-93-DE   Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States
Gene activated A-galactosidase A
   Shire Human Genetic Therapies, Inc.
      2022   Phase 3   EUCTR2018-004689-32-FI   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-SE   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PT   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PL   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-GR   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
Gene activated human glucocerebrosidase
   SHIRE HUMAN GENETIC THERAPIES, INC.
      2008   -   EUCTR2007-002840-21-IT   Italy;Spain;United Kingdom
   Shire Human Genetic Therapies Inc
      2007   Phase 3   EUCTR2007-002840-21-ES   Italy;Spain;United Kingdom
      2007   Phase 2;Phase 3   EUCTR2006-006304-11-GB   Germany;Italy;Spain;United Kingdom
      2007   Phase 2;Phase 3   EUCTR2006-006304-11-ES   Germany;Italy;Spain;United Kingdom
      -   Phase 2;Phase 3   EUCTR2006-006304-11-DE   Germany;Italy;Spain;United Kingdom
   Shire Human Genetic Therapies, Inc.
      2008   -   EUCTR2008-001965-27-ES   Spain;United Kingdom
      2007   -   EUCTR2007-002840-21-GB   Italy;Spain;United Kingdom
Gene activated human glucocerebrosidase, velaglucerase alfa
   Shire Human Genetic Therapies, Inc.
      2016   Phase 4   EUCTR2015-001578-17-GB   Canada;Germany;Israel;Spain;United Kingdom;United States
      2016   Phase 4   EUCTR2015-001578-17-ES   Canada;Germany;Israel;Italy;Spain;United Kingdom;United States
      2016   Phase 4   EUCTR2015-001578-17-DE   Germany;India;Israel;Italy;Spain;Turkey;United Kingdom;United States
      2008   Phase 3   EUCTR2008-001965-27-GB   Spain;United Kingdom
Gene therapy
   AVROBIO
      2023   Phase 2/Phase 3   NCT05815004   -
GENE-activated human glucocerebrosidase
   SHIRE HUMAN GENETIC THERAPIES, INC.
      2008   -   EUCTR2006-006304-11-IT   Italy;Spain;United Kingdom
   Shire Human Genetic Therapies Inc
      2007   Phase 2;Phase 3   EUCTR2006-006304-11-GB   Germany;Italy;Spain;United Kingdom
      2007   Phase 2;Phase 3   EUCTR2006-006304-11-ES   Germany;Italy;Spain;United Kingdom
      -   Phase 2;Phase 3   EUCTR2006-006304-11-DE   Germany;Italy;Spain;United Kingdom
   Shire Human Genetic Therapies, Inc.
      2008   -   EUCTR2008-001965-27-ES   Spain;United Kingdom
GENE-activated human glucocerebrosidase 200U/vial
   Shire Human Genetic Therapies, Inc.
      2008   Phase 3   EUCTR2008-001965-27-GB   Spain;United Kingdom
GENE-activated human glucocerebrosidase 400U/vial
   Shire Human Genetic Therapies
      -   -   EUCTR2012-003427-38-Outside-EU/EEA   Egypt;India;Tunisia
   Shire Human Genetic Therapies, Inc.
      2008   Phase 3   EUCTR2008-001965-27-GB   Spain;United Kingdom
General and neurological examination
   Baylor Research Institute
      2018   -   NCT04002531   United States
Genetic analyses
   Hospital St. Joseph, Marseille, France
      2010   -   NCT02650219   France
Genetic HMI-203
   Homology Medicines, Inc
      2022   Phase 1   NCT05238324   Canada;United States
Genistein aglycone
   Manchester University NHS Foundation Trust
      2014   Phase 3   EUCTR2013-001479-18-GB   United Kingdom
GENZ-112638
   Genzyme Europe B.V.
      2011   Phase 3   EUCTR2009-015811-42-PT   Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States
      2011   -   EUCTR2009-015811-42-GR   Austria;Greece;Portugal;Sweden
      2011   Phase 3   EUCTR2009-015811-42-AT   Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States
      2010   Phase 3   EUCTR2009-015811-42-SE   Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States
      2010   -   EUCTR2008-005223-28-ES   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2010   -   EUCTR2008-005223-28-DE   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2010   -   EUCTR2008-005222-37-BG   Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States
      2009   Phase 3   EUCTR2008-005223-28-NL   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005223-28-GB   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2008-005223-28-FR   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   -   EUCTR2008-005223-28-CZ   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   -   EUCTR2008-005222-37-NL   Bulgaria;Netherlands;United Kingdom
      2009   Phase 3   EUCTR2008-005222-37-GB   Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States
   Genzyme Europe BV
      2010   -   EUCTR2008-005223-28-IT   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2006   -   EUCTR2005-004732-42-IT   Italy
   Sanofi
      2009   Phase 1   NCT06143904   -
GENZ-682452-AA
   Genzyme Corporation
      2014   Phase 2   EUCTR2013-005324-41-GB   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
      2014   Phase 2   EUCTR2013-005324-41-CZ   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
GENZ-682452-AU
   GENZYME CORPORATION
      2020   Phase 3   EUCTR2019-002375-34-IT   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
   Genzyme Corporation
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-FR   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 3   EUCTR2019-002375-34-AT   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-PT   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-GB   Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-DE   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Türkiye;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-CZ   Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2016   Phase 2   EUCTR2014-002550-39-DE   Germany;Japan;United Kingdom;United States
      2015   Phase 2   EUCTR2014-004995-49-PL   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
      2015   Phase 2   EUCTR2014-004995-49-GB   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
      2015   Phase 2   EUCTR2014-004995-49-FR   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-003120-17-SE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      -   Phase 3   EUCTR2019-002375-34-FR   Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
GLA gene
   Chiayi Christian Hospital
      2016   -   NCT03596398   -
Glucocerebrosidase
   National Center for Research Resources (NCRR)
      1999   Phase 2   NCT00004293   United States
   Pfizer
      2007   Phase 3   NCT00376168   Canada;Chile;Israel;Italy;South Africa;Spain;United Kingdom;United States
      2006   -   NCT00962260   Israel;United States
   Protalix
      2005   Phase 1   NCT00258778   Israel
Glucocerebrosidase umano GENE-attivato
   SHIRE HUMAN GENETIC THERAPIES, INC
      2018   Phase 4   EUCTR2015-001578-17-IT   Germany;Israel;Italy;Spain;United Kingdom;United States
GNR-055
   AO GENERIUM
      2020   Phase 1   NCT04539340   Russian Federation
GNR-055 1.0-2.0-3.0 MG/KG
   AO GENERIUM
      2021   Phase 2/Phase 3   NCT05208281   Russian Federation
GNR-055 2.0 MG/KG
   AO GENERIUM
      2021   Phase 2/Phase 3   NCT05208281   Russian Federation
GNR-055 3.0 MG/KG
   AO GENERIUM
      2021   Phase 2/Phase 3   NCT05208281   Russian Federation
GR181413
   GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
      2012   -   EUCTR2011-004800-40-IT   Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States
   GlaxoSmithKline Research & Development Limited
      2012   Phase 3   EUCTR2011-004800-40-ES   Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
GR181413A
   Amicus Therapeutics, Inc.
      2014   Phase 3   EUCTR2011-004800-40-AT   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2013   Phase 3   EUCTR2011-004800-40-BE   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2012   Phase 3   EUCTR2011-004800-40-GB   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2012   Phase 3   EUCTR2011-004800-40-DK   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
   GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
      2012   -   EUCTR2011-004800-40-IT   Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States
   GlaxoSmithKline Research & Development Limited
      2012   Phase 3   EUCTR2011-004800-40-ES   Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
GR181413A/AT1001 capsule
   Amicus Therapeutics
      2011   Phase 1   NCT01853852   Australia
GR181413A/AT1001 solution
   Amicus Therapeutics
      2011   Phase 1   NCT01853852   Australia
Growth hormone
   University of Texas Southwestern Medical Center
      2006   -   NCT00286689   United States
GSK2788723
   GlaxoSmithKline
      2012   -   NCT01602601   Japan
GZ/SAR402671
   Genzyme, a Sanofi Company
      2015   Phase 2   NCT02489344   France;Poland;Russian Federation;United Kingdom;United States
      2014   Phase 2   NCT02228460   Czech Republic;Czechia;France;Poland;Russian Federation;United Kingdom;United States
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2023   Phase 3   EUCTR2021-005402-10-IT   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002350-90-IT   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
   Sanofi-aventis recherche & développement
      2022   Phase 3   EUCTR2021-005402-10-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005402-10-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Türkiye;United Kingdom;United States
GZ385660
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000301-37-NL   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2019   Phase 3   EUCTR2016-000301-37-GB   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-SE   Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-IT   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-ES   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
GZ402665
   GENZYME CORPORATION
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-IT   Argentina;Australia;Brazil;Chile;France;Germany;Israel;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
   Genzyme Corporation
      2019   Phase 2   EUCTR2013-000051-40-BE   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2018   Phase 2   EUCTR2013-000051-40-DE   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-PT   Argentina;Australia;Belgium;Brazil;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-NL   Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2016   Phase 2   EUCTR2013-000051-40-IT   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2;Phase 3   EUCTR2015-000371-26-GB   Argentina;Australia;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   -   EUCTR2015-000371-26-ES   Argentina;Australia;Brazil;Chile;France;Germany;Israel;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   Phase 2;Phase 3   EUCTR2015-000371-26-DE   Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-IT   Brazil;Chile;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-GB   Brazil;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-DE   Brazil;France;Germany;Italy;United Kingdom;United States
      2014   -   EUCTR2010-023953-12-IT   Brazil;Chile;France;Germany;Italy;Tunisia;United Kingdom;United States
      2013   Phase 2   EUCTR2013-000051-40-GB   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2013   -   EUCTR2010-023953-12-GB   Brazil;Chile;France;Germany;Italy;Tunisia;United Kingdom;United States
      2013   -   EUCTR2010-023953-12-FR   Brazil;Chile;France;Germany;Italy;Tunisia;United Kingdom;United States
      -   Phase 2   EUCTR2014-003198-40-FR   Brazil;Chile;France;Germany;Italy;United Kingdom;United States
   Genzyme, a Sanofi Company
      2015   Phase 2/Phase 3   NCT02004691   Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2013   Phase 2   NCT02004704   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
   Sanofi
      2021   -   NCT04877132   -
   Tanaka Tomoyuki
      2021   Phase 2-3   JPRN-jRCT2031210279   -
GZ402671
   GENZYME CORPORATION
      2020   Phase 3   EUCTR2019-002375-34-IT   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
   Genzyme Corporation
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-FR   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 3   EUCTR2019-002375-34-AT   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-PT   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-GB   Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-DE   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Türkiye;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-CZ   Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2016   Phase 2   EUCTR2014-002550-39-DE   Germany;Japan;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-003120-17-SE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      -   Phase 3   EUCTR2019-002375-34-FR   Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
   Genzyme, a Sanofi Company
      2017   Phase 2   NCT02843035   Germany;Japan;United Kingdom;United States
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002350-90-IT   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
   Sanofi
      2022   Phase 3   NCT05280548   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   NCT05206773   Argentina;Australia;Austria;Brazil;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Japan;Mexico;Norway;Poland;Romania;Switzerland;Turkey;United Kingdom;United States
   Sanofi-aventis recherche & développement
      -   Phase 3   EUCTR2021-005402-10-FR   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
   Tanaka Tomoyuki
      2022   Phase 3   JPRN-jRCT2071220022   China;Italy;Japan;Republic of Korea
      2022   Phase 3   JPRN-jRCT2021220001   Argentina;Austria;Brazil;Canada;China;Denmark;Finland;Germany;Greece;Italy;Japan;Mexico;Norway;Poland;Romania;United Kingdom;United States
GZ402671 / SAR402671
   Genzyme Corporation
      2014   Phase 2   EUCTR2013-005324-41-GB   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
      2014   Phase 2   EUCTR2013-005324-41-CZ   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
Hematopoetic stem cell transplantation
   University of Pittsburgh
      2012   -   NCT03333200   United States
Hematopoietic stem cell infusion
   Talaris Therapeutics Inc.
      2011   Phase 1/Phase 2   NCT01372228   United States
Hematopoietic stem cell transplantation
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
      2006   Phase 2   NCT00383448   United States
HeparáN N sulfatasa humana recombinante
   Shire Human Genetic Therapies, Inc
      2014   Phase 2   EUCTR2013-003450-24-ES   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
HGT-1110
   Shire
      2013   Phase 1/Phase 2   NCT01887938   Argentina;Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Japan
   Shire Human Genetics Therapies Inc
      2012   Phase 1;Phase 2   EUCTR2012-003775-20-FR   Argentina;Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;United Kingdom
      2012   Phase 1;Phase 2   EUCTR2011-002044-28-DE   Australia;Brazil;Denmark;France;Germany;Japan
      2011   Phase 1;Phase 2   EUCTR2011-002044-28-DK   Australia;Brazil;Denmark;France;Germany;Japan
HGT-1111
   Shire
      2008   Phase 2   NCT00681811   Denmark
   Shire Human Genetic Therapies Inc
      2010   Phase 2   EUCTR2008-000084-41-BE   Belgium;Denmark;France;Italy
      2009   Phase 2   EUCTR2008-000084-41-IT   Belgium;Denmark;France;Italy
      2009   Phase 2   EUCTR2008-000084-41-FR   Belgium;Denmark;France;Italy
      2008   Phase 2   EUCTR2008-000084-41-DK   Belgium;Denmark;France;Italy
HGT-1410
   SHIRE HUMAN GENETIC THERAPIES, INC
      2015   Phase 2   EUCTR2014-003960-20-IT   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire
      2015   Phase 2   NCT02350816   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetic Therapies, Inc
      2014   Phase 2   EUCTR2013-003450-24-NL   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003450-24-IT   France;Germany;Italy;Netherlands;Spain;United Kingdom
      2014   Phase 2   EUCTR2013-003450-24-GB   Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003450-24-ES   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003450-24-DE   Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2011   Phase 1;Phase 2   EUCTR2010-021348-16-NL   Netherlands;United Kingdom
      2011   Phase 1;Phase 2   EUCTR2010-021348-16-GB   Netherlands;United Kingdom
      -   Phase 2   EUCTR2013-003450-24-FR   Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetic Therapies, Inc.
      2015   Phase 2   EUCTR2014-003960-20-NL   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003960-20-GB   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003960-20-DE   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetic Therapies, Inc. (Shire HGT)
      2010   Phase 1;Phase 2   EUCTR2009-015984-15-GB   United Kingdom
   Shire human Genetic Therapies, Inc.
      2015   Phase 2   EUCTR2014-003960-20-ES   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      -   Phase 2   EUCTR2014-003960-20-FR   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
HGT-2310
   Shire HGT Inc
      2013   Phase 2;Phase 3   EUCTR2013-002885-38-GB   Argentina;Australia;Canada;Colombia;France;Germany;Mexico;Spain;United Kingdom;United States
      2013   Phase 2;Phase 3   EUCTR2013-002885-38-ES   Argentina;Mexico;Spain;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2013-002885-38-FR   Argentina;Australia;France;Germany;Mexico;Spain;United Kingdom;United States
   Shire Human Genetic Therapies, Inc.
      2015   Phase 2;Phase 3   EUCTR2014-004143-13-GB   Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States
      2015   -   EUCTR2014-004143-13-ES   Argentina;Canada;Colombia;Mexico;Spain;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2014-004143-13-FR   Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States
High dose AT-GTX-502
   Amicus Therapeutics
      2018   Phase 1/Phase 2   NCT03770572   United States
HM15421/GC1134A
   GC Biopharma Corp
      2025   Phase 1/Phase 2   NCT06858397   Argentina;Korea, Republic of;United States
Hucns-SC
   StemCells, Inc.
      2010   Phase 1   NCT01238315   United States
      2006   Phase 1   NCT00337636   United States
Human glucocerebrosidase cdna
   National Institute of Neurological Disorders and Stroke (NINDS)
      1988   Phase 1   NCT00001234   United States
Human glucocerebrosidase gene into autologous peripheral blood stem cells
   National Center for Research Resources (NCRR)
      1999   Phase 1   NCT00004294   United States
Human N-sulfoglucosamine sulfohydrolase fused TO A humanised monoclonal antibody targeting human transferrin receptor
   JCR Pharmaceuticals Co., Ltd.
      2023   Phase 1;Phase 2   EUCTR2022-002314-17-DE   Germany
Human placental derived stem cell
   New York Medical College
      2013   Phase 1   NCT01586455   United States
Hunterase
   Green Cross Corporation
      2012   Phase 3   NCT01645189   Korea, Republic of
Hydroxypropyl-BETA-cyclodextrin
   CTD Holdings, Inc.
      2016   Phase 1;Phase 2   EUCTR2015-005761-23-GB   Italy;Sweden;United Kingdom
   Cyclo Therapeutics, Inc.
      2017   Phase 1   NCT02939547   United States
      2017   Phase 1/Phase 2   NCT02912793   Israel;Sweden;United Kingdom
      2017   Phase 1;Phase 2   EUCTR2015-005761-23-SE   Italy;Sweden;United Kingdom
Hydroxypropyl-ß-cyclodextrin
   Cyclo Therapeutics, Inc.
      2019   Phase 1   NCT03893071   United States
Hydroxyurea
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States
I2S
   Shire Human Genetic Therapies INC.
      2005   -   EUCTR2004-002743-27-SE   Germany;Italy;Spain;Sweden;United Kingdom
      2004   -   EUCTR2004-002743-27-DE   Germany;Italy;Spain;Sweden;United Kingdom
   Shire Human Genetic Therapies, Inc. (Shire HGT)
      -   Phase 4   EUCTR2007-006044-22-PL   Poland
   TKT INC
      2005   -   EUCTR2004-002743-27-IT   Germany;Italy;Spain;Sweden;United Kingdom
   TKT Inc.
      2005   Phase 3   EUCTR2004-002743-27-GB   Germany;Italy;Spain;Sweden;United Kingdom
      2005   Phase 3   EUCTR2004-002743-27-ES   Germany;Italy;Spain;Sweden;United Kingdom
I2S-IT
   Shire HGT Inc
      -   -   EUCTR2011-000212-25-Outside-EU/EEA   Canada;United States
   Shire HGT, Inc
      2011   Phase 1;Phase 2   EUCTR2010-020048-36-GB   United Kingdom;United States
IB1001
   IntraBio Inc
      2019   Phase 2   NCT03759665   Germany;Spain;United Kingdom;United States
      2019   Phase 2   NCT03759639   Germany;Slovakia;Spain;United Kingdom;United States
   IntraBio Ltd
      2022   Phase 3   EUCTR2021-005356-10-SK   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-NL   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-DE   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-CZ   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004406-25-GB   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004406-25-ES   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004406-25-DE   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-SK   Germany;Slovakia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-GB   Germany;Slovakia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-ES   Germany;Slovakia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-DE   Germany;Slovakia;Spain;United Kingdom;United States
Iduronate-2-sulfatase enzyme replacement therapy
   Shire
      2003   Phase 2/Phase 3   NCT00069641   Brazil;Germany;United Kingdom;United States
Iduronate-2-sulfatase fused TO A FC polypeptide that binds TO THE human transferrin receptor
   Denali Therapeutics Inc.
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-SE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-NL   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-IT   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-FR   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-CZ   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-BE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2021   Phase 1;Phase 2   EUCTR2019-004909-27-NL   Germany;Italy;Netherlands;United Kingdom;United States
      2020   Phase 1;Phase 2   EUCTR2019-004909-27-GB   Germany;Italy;Netherlands;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-005200-35-DE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
Idursulfasa
   Shire HGT Inc
      2013   Phase 2;Phase 3   EUCTR2013-002885-38-ES   Argentina;Mexico;Spain;United Kingdom;United States
Idursulfase
      2007   -   EUCTR2007-001453-26-NL   Netherlands
   Denali Therapeutics Inc.
      2022   Phase 2/Phase 3   NCT05371613   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czechia;France;Germany;Italy;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-SE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-NL   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-IT   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-FR   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-CZ   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-BE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-005200-35-DE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
   GlaxoSmithKline
      2012   -   NCT01602601   Japan
   JCR Pharmaceuticals Co., Ltd.
      2023   Phase 3   EUCTR2020-003200-14-PL   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003200-14-DE   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Türkiye;United Kingdom;United States
   Shire
      2009   Phase 1/Phase 2   NCT00920647   United Kingdom;United States
      2008   -   NCT00882921   Brazil;United Kingdom;United States
      2007   Phase 4   NCT00607386   Brazil;Poland;Taiwan
      2004   Phase 2/Phase 3   NCT00630747   Brazil;Canada;France;Germany;Italy;Romania;Spain;Sweden;United Kingdom;United States
   Shire Human Genetic Therapies INC.
      2005   -   EUCTR2004-002743-27-SE   Germany;Italy;Spain;Sweden;United Kingdom
      2004   -   EUCTR2004-002743-27-DE   Germany;Italy;Spain;Sweden;United Kingdom
   Shire Human Genetic Therapies, Inc. (Shire HGT)
      -   Phase 4   EUCTR2007-006044-22-PL   Poland
   Shire Human Genetic Therapies, Inc. (Shire), a wholly owned subsidiary of Takeda Pharmaceutical Company Limited
      2016   Phase 4   EUCTR2014-004804-31-DE   Costa Rica;Dominican Republic;Germany;Malaysia;Oman;Philippines;Saudi Arabia;Serbia;Thailand;United States;Viet Nam
   TKT INC
      2005   -   EUCTR2004-002743-27-IT   Germany;Italy;Spain;Sweden;United Kingdom
   TKT Inc.
      2005   Phase 3   EUCTR2004-002743-27-GB   Germany;Italy;Spain;Sweden;United Kingdom
      2005   Phase 3   EUCTR2004-002743-27-ES   Germany;Italy;Spain;Sweden;United Kingdom
Idursulfase (I2S)-IT
   Shire HGT, Inc
      2011   Phase 1;Phase 2   EUCTR2010-020048-36-GB   United Kingdom;United States
Idursulfase beta
   Green Cross Corporation
      2016   Phase 2   NCT02663024   -
   Yanagi Komei
      2020   Phase 3   JPRN-jRCT2031220250   -
Idursulfase(12S)-IT
   Shire HGT Inc
      2012   Phase 1;Phase 2   EUCTR2011-000212-25-GB   Canada;United Kingdom;United States
Idursulfase(I2S)-IT
   Shire HGT Inc
      -   -   EUCTR2011-000212-25-Outside-EU/EEA   Canada;United States
Idursulfase-IT
   Shire
      2015   Phase 2/Phase 3   NCT02412787   Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States
      2014   Phase 2/Phase 3   NCT02055118   Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States
      2010   Phase 1/Phase 2   NCT01506141   Canada;United Kingdom;United States
   Shire HGT Inc
      2013   Phase 2;Phase 3   EUCTR2013-002885-38-GB   Argentina;Australia;Canada;Colombia;France;Germany;Mexico;Spain;United Kingdom;United States
      2013   Phase 2;Phase 3   EUCTR2013-002885-38-ES   Argentina;Mexico;Spain;United Kingdom;United States
      2012   Phase 1;Phase 2   EUCTR2011-000212-25-GB   Canada;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2013-002885-38-FR   Argentina;Australia;France;Germany;Mexico;Spain;United Kingdom;United States
      -   -   EUCTR2011-000212-25-Outside-EU/EEA   Canada;United States
   Shire HGT, Inc
      2011   Phase 1;Phase 2   EUCTR2010-020048-36-GB   United Kingdom;United States
   Shire Human Genetic Therapies, Inc.
      2015   Phase 2;Phase 3   EUCTR2014-004143-13-GB   Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States
      2015   -   EUCTR2014-004143-13-ES   Argentina;Canada;Colombia;Mexico;Spain;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2014-004143-13-FR   Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States
   Takeda
      2024   Phase 2/Phase 3   NCT06031259   Canada;France
      2023   -   NCT05795361   Australia;Mexico;Spain;United Kingdom;United States
Idursulfasi
   Denali Therapeutics Inc.
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-IT   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
   JCR Pharmaceutical Co., Ltd.
      2023   Phase 3   EUCTR2020-003200-14-IT   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Turkey;United Kingdom;United States
IMD preparative regimen
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States
Imiglucerasa
   INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD
      2008   -   EUCTR2007-007164-20-ES   Spain
Imiglucerase
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000301-37-NL   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2019   Phase 3   EUCTR2016-000301-37-GB   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-SE   Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-IT   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
      2018   Phase 3   EUCTR2016-000301-37-ES   Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom
   Genzyme Europe B.V.
      2010   -   EUCTR2008-005223-28-DE   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2008-005223-28-NL   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005223-28-GB   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2008-005223-28-FR   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   -   EUCTR2008-005223-28-CZ   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
   Genzyme Europe BV
      2010   -   EUCTR2008-005223-28-IT   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
   Genzyme, a Sanofi Company
      2017   Phase 2   NCT02843035   Germany;Japan;United Kingdom;United States
      2009   Phase 3   NCT00943111   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      1997   Phase 4   NCT00365131   United States
   ISTITUTO GIANNINA GASLINI
      2009   -   EUCTR2007-005516-61-IT   Italy
   SHIRE HUMAN GENETIC THERAPIES, INC.
      2008   -   EUCTR2007-002840-21-IT   Italy;Spain;United Kingdom
   Sanofi
      2022   Phase 3   NCT05222906   Argentina;Canada;China;France;Germany;Hungary;Italy;Japan;Turkey;United Kingdom;United States
   Sanofi-aventis recherche & développement
      2022   Phase 3   EUCTR2021-005402-10-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005402-10-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Türkiye;United Kingdom;United States
   Shire
      2008   Phase 3   NCT00553631   Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom;United States
   Shire Human Genetic Therapies Inc
      2007   Phase 3   EUCTR2007-002840-21-ES   Italy;Spain;United Kingdom
   Shire Human Genetic Therapies, Inc.
      2007   -   EUCTR2007-002840-21-GB   Italy;Spain;United Kingdom
   Tanaka Tomoyuki
      2023   Phase 3   JPRN-jRCT2021220029   Argentina;Canada;China;France;Germany;Hungary;Japan;United States
   University Research Foundation for Lysosomal Storage Diseases, Inc.
      2010   -   NCT01136304   United States
Imiglucerase GZ437843
   Sanofi
      2018   Phase 3   NCT03485677   Argentina;Canada;France;Italy;Japan;Russian Federation;Spain;Sweden;Turkey;United Kingdom
Imiglucerasi
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2023   Phase 3   EUCTR2021-005402-10-IT   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
   SHIRE HUMAN GENETIC THERAPIES, INC.
      2008   -   EUCTR2007-002840-21-IT   Italy;Spain;United Kingdom
Imiglucérase (drug) pharmacokinetics
   University Hospital, Clermont-Ferrand
      2012   Phase 2   NCT01951989   France
Inactive reminder capsules
   Amicus Therapeutics, Inc.
      2015   Phase 3   EUCTR2014-002701-38-GB   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2015   Phase 3   EUCTR2014-002701-38-DK   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2015   Phase 3   EUCTR2014-002701-38-BE   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2014   Phase 3   EUCTR2014-002701-38-AT   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2012   -   EUCTR2010-022636-37-DE   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   Phase 3   EUCTR2010-022636-37-GR   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-GB   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-DK   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-BE   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-AT   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
Inactive reminder product
   Amicus Therapeutics, Inc.
      2010   -   EUCTR2009-013459-31-DK   Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2009-013459-31-GB   Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States
INN NOT available
   Genzyme Europe B.V.
      2010   -   EUCTR2008-005223-28-ES   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
Innozide
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Intracerebral administration OF aavrh.10cuarsa
   Institut National de la Santé Et de la Recherche Médicale, France
      2014   Phase 1/Phase 2   NCT01801709   France
Intrathecal recombinant human alpha iduronidase
   agnes chen
      2014   -   NCT02232477   United States
Intraventricular access device
   BioMarin Pharmaceutical
      2016   Phase 2   NCT02678689   Germany;Italy;United Kingdom;United States
      2015   Phase 1/Phase 2   NCT02485899   Germany;Italy;United Kingdom;United States
Iohexol
   Genzyme, a Sanofi Company
      2013   -   NCT01839526   Argentina;Austria;Belgium;Brazil;Canada;Finland;France;Hungary;Norway;Poland;Spain;Taiwan;United Kingdom;United States
      2012   Phase 1   EUCTR2012-001966-14-ES   Canada;Netherlands;Norway;Spain;United Kingdom;United States
IRT laronidase
   Masonic Cancer Center, University of Minnesota
      2008   Phase 1   NCT00638547   United States
Isaralgagene civaparvovec
   Sangamo Therapeutics, Inc.
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-DE   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
Isofagamine tartrate
   Amicus Therapeutics, Inc.
      2007   -   EUCTR2007-003359-35-GB   Germany;United Kingdom
      2007   -   EUCTR2007-003359-35-DE   Germany;United Kingdom
ISU302
   ISU Abxis Co., Ltd.
      2011   Phase 3   NCT02770625   -
      2011   Phase 3   NCT01161914   -
      2010   Phase 1   NCT01881633   -
IV migalastat HCL
   Amicus Therapeutics
      2014   Phase 1   NCT02082327   Netherlands
IVA 336
   Inventiva S.A.
      2019   Phase 2   EUCTR2017-002158-35-PT   France;Germany;Portugal;United Kingdom
      2018   Phase 2   EUCTR2017-002158-35-DE   France;Germany;Portugal;United Kingdom
      2017   Phase 2   EUCTR2017-002158-35-GB   France;Germany;Portugal;United Kingdom
      -   Phase 2   EUCTR2017-002158-35-FR   France;Germany;Portugal;United Kingdom
Izcargo
   Ibaraki Ryo
      2021   Phase 4   JPRN-jRCT2031210126   -
   JCR Pharmaceuticals Co., Ltd.
      2023   Phase 3   EUCTR2020-003200-14-PL   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Türkiye;United Kingdom;United States
JR-141
   Ibaraki Ryo
      2021   Phase 4   JPRN-jRCT2031210126   -
   JCR Pharmaceutical Co., Ltd.
      2023   Phase 3   EUCTR2020-003200-14-IT   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Turkey;United Kingdom;United States
   JCR Pharmaceuticals Co., Ltd.
      2023   Phase 3   NCT05594992   Argentina;Brazil;France;Germany;Italy;Spain;United States
      2023   Phase 3   EUCTR2020-003200-14-PL   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003200-14-FR   Brazil;France;Germany;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003200-14-DE   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Türkiye;United Kingdom;United States
      2019   Phase 2/Phase 3   NCT04348136   Japan
      2019   Phase 2   NCT03708965   Brazil
      2018   Phase 2/Phase 3   NCT03568175   Japan
      2018   Phase 2   NCT03359213   Brazil
      2017   Phase 1/Phase 2   NCT03128593   Japan
JR-141 or idursulfase
   JCR Pharmaceuticals Co., Ltd.
      2022   Phase 3   NCT04573023   Argentina;Brazil;Colombia;France;Germany;Israel;Italy;Poland;Spain;Turkey;United Kingdom;United States
JR-171
   JCR Pharmaceuticals Co., Ltd.
      2021   Phase 1/Phase 2   NCT04453085   Brazil;Japan;United States
      2020   Phase 1/Phase 2   NCT04227600   Brazil;Japan;United States
JR-441
   JCR Pharmaceuticals Co., Ltd.
      2023   Phase 1/Phase 2   NCT06095388   Germany
      2023   Phase 1;Phase 2   EUCTR2022-002314-17-DE   Germany
JR-446
   JCR Pharmaceuticals Co., Ltd.
      2024   Phase 1/Phase 2   NCT06488924   Japan
JWK008 single intravenous infusion administration
   West China Hospital
      2024   Phase 1   NCT06519552   China
Kanuma
   Alexion Pharmaceuticals, Inc.
      2015   Phase 3   EUCTR2011-002750-31-CZ   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-GB   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-DE   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      -   Phase 3   EUCTR2011-002750-31-PL   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
Ketogenic diet
   University of Minnesota
      2015   Phase 4   NCT02030015   United States
Kidney transplant
   Alice Bertaina
      2023   Phase 1/Phase 2   NCT05508009   United States
Laboratory tests
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Lamazym
   Chiesi Farmaceutici S.p.A.
      2016   Phase 2   NCT02998879   Austria;Denmark;France;Germany;Italy
      2015   Phase 3   EUCTR2013-000336-97-DK   Denmark;France
      2013   Phase 3   NCT01908725   Denmark
      2013   Phase 3   NCT01908712   France
   Zymenex A/S
      2015   Phase 3   NCT02478840   Denmark
      2015   -   EUCTR2014-003950-15-DK   Denmark
      2013   Phase 3   EUCTR2013-000336-97-FR   Denmark;France
      2013   -   EUCTR2012-000979-17-SE   Belgium;Denmark;Germany;Spain;Sweden
      2013   Phase 3   EUCTR2012-000979-17-ES   Belgium;Denmark;Germany;Spain;Sweden
      2013   -   EUCTR2012-000979-17-BE   Belgium;Denmark;Germany;Spain;Sweden
      2012   Phase 3   NCT01681953   Denmark;France;Germany;Poland;United Kingdom
      2012   Phase 2   NCT01681940   Belgium;Denmark;Spain;United Kingdom
      2012   -   EUCTR2012-000979-17-DK   Belgium;Denmark;Germany;Spain;Sweden
      2012   -   EUCTR2012-000979-17-DE   Belgium;Denmark;Germany;Spain;Sweden
      2012   -   EUCTR2011-004355-40-GB   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   -   EUCTR2011-004355-40-ES   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   Phase 2   EUCTR2011-004355-40-DK   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   -   EUCTR2011-004355-40-DE   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   -   EUCTR2011-004355-40-BE   Belgium;Denmark;Germany;Spain;United Kingdom
      2011   Phase 2   NCT01285700   Denmark
      2010   Phase 1   NCT01268358   -
      2010   -   EUCTR2010-022085-26-DK   Denmark
      2010   -   EUCTR2010-022084-36-DK   Denmark
Laronidase
      2007   -   EUCTR2007-001453-26-NL   Netherlands
   Cinnagen
      2022   Phase 3   NCT06406153   Iran, Islamic Republic of
   Genzyme Europe B. V
      -   Phase 4   EUCTR2023-001027-16-Outside-EU/EEA   China
   Genzyme Europe BV
      2010   -   EUCTR2007-007003-33-IT   Italy
   Genzyme, a Sanofi Company
      2021   Phase 4   NCT05134571   China
      2010   Phase 4   NCT00418821   Italy;United States
      2008   Phase 1/Phase 2   NCT00741338   Brazil;Russian Federation;Ukraine
      2004   Phase 4   NCT00144768   United States
      -   -   EUCTR2015-000585-61-Outside-EU/EEA   United States
      -   -   EUCTR2007-001163-30-Outside-EU/EEA   Brazil;Russian Federation
   HUS, Hospital for Children and Adolescents
      2007   -   EUCTR2006-005216-27-FI   Finland
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01572636   United States
      2012   Phase 1   NCT01173016   United States
   Patricia I. Dickson, M.D.
      2009   -   NCT00852358   United States
      2008   Phase 1   NCT00786968   Finland;United States
      2005   Phase 1   NCT00215527   Finland;United States
   University of California, San Francisco
      2021   Phase 1   NCT04532047   United States
Laronidase ERT
   Masonic Cancer Center, University of Minnesota
      2004   Phase 2   NCT00176891   United States
Laronidase therapy and A stem cell transplant
   Masonic Cancer Center, University of Minnesota
      2022   -   NCT05634512   United States
Ledipasvir/sofosbuvir
   Mansoura University Children Hospital
      2018   Phase 4   NCT03721627   Egypt
Lenograstim
   OSPEDALE SAN RAFFAELE
      2018   Phase 1   EUCTR2017-002430-23-IT   Italy
Lentivirus alpha-GAL A transduced stem cells
   University Health Network, Toronto
      2016   Phase 1   NCT02800070   Canada
Lepunafusp alfa
   JCR Pharmaceuticals Co., Ltd.
      2020   Phase 1/Phase 2   NCT04227600   Brazil;Japan;United States
Leucovorin
   The Hospital for Sick Children
      2009   Phase 1/Phase 2   NCT01102686   Canada
Lisinopril
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Lopace
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Loratadine
   Sanofi
      2023   Phase 4   NCT06019728   United States
Losartan
   Hospital de Clinicas de Porto Alegre
      2018   Phase 2   NCT03632213   Brazil
Losartan and other angiotensin receptor blockers
   University of Alabama at Birmingham
      2007   -   NCT00446862   Germany;Slovenia;United States
LOW dose AT-GTX-502
   Amicus Therapeutics
      2018   Phase 1/Phase 2   NCT03770572   United States
Lucerastat
   IDORSIA PHARMACEUTICALS LTD
      2020   Phase 3   EUCTR2018-002210-12-IT   Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-IT   Australia;Austria;Belgium;Canada;Finland;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
   Idorsia Pharmaceuticals Japan Ltd
      2019   Phase 3   JPRN-jRCT2080224890   Japan
      2019   Phase 3   JPRN-jRCT2080224659   Japan
   Idorsia Pharmaceuticals Ltd
      2021   Phase 3   EUCTR2018-002210-12-NO   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2021   Phase 3   EUCTR2018-002210-12-FR   Austria;Belgium;Canada;Czech Republic;France;Germany;Netherlands;Poland;United Kingdom;United States
      2020   Phase 3   EUCTR2018-002210-12-BE   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-NO   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-IE   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-ES   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Russian Federation;Spain;Switzerland;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-NL   Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-GB   Australia;Austria;Belgium;Canada;Germany;Ireland;Netherlands;Poland;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-DE   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-AT   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2017-003369-85-NL   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2018   Phase 3   EUCTR2017-003369-85-GB   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2018   Phase 3   EUCTR2017-003369-85-AT   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
   Idorsia Pharmaceuticals Ltd.
      2018   Phase 3   NCT03737214   Australia;Austria;Belgium;Canada;Czechia;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2018   Phase 3   NCT03425539   Australia;Austria;Belgium;Canada;Czechia;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2015   Phase 1   NCT02930655   Germany
Lumbar puncture
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
LY-M001
   Shanghai Jiao Tong University School of Medicine
      2024   Early Phase 1   NCT06528080   China
LY-M001 injection
   He Huang
      2023   -   NCT06162338   China
   Lingyi Biotech Co., Ltd.
      2024   Phase 1/Phase 2   NCT06818838   China
LY3884961
   Prevail Therapeutics
      2022   Phase 1/Phase 2   NCT05487599   Australia;Germany;Spain;United Kingdom;United States
      2021   Phase 1/Phase 2   NCT04411654   United Kingdom;United States
Lymphocyte gene therapy
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      1996   Phase 1/Phase 2   NCT00004454   United States
LYS-GM101
   LYSOGENE
      2021   Phase 1/Phase 2   NCT04273269   France;United Kingdom;United States
   Lysogene
      -   Phase 1;Phase 2   EUCTR2019-004949-32-FR   France;United Kingdom;United States
LYS-SAF302
   Lysogene SA
      2019   Phase 2;Phase 3   EUCTR2018-000195-15-NL   France;Germany;Netherlands;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-000195-15-GB   France;Germany;Netherlands;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-000195-15-DE   France;Germany;Netherlands;United Kingdom;United States
      2018   Phase 2;Phase 3   EUCTR2018-000195-15-FR   France;Germany;Netherlands;United Kingdom;United States
Lysodase
   National Institute of Mental Health (NIMH)
      1993   Phase 1   NCT00001410   United States
Mabthera - 2 fiale 100 MG 10 ML
   OSPEDALE SAN RAFFAELE
      2018   Phase 1   EUCTR2017-002430-23-IT   Italy
Magnetic resonance imaging
   University Hospital, Rouen
      2017   -   NCT02956954   France
Mechanical stimulation with VON frey filaments
   Universitaire Ziekenhuizen Leuven
      2014   Phase 0   NCT02533076   Belgium
Medication TO suppress THE immune system
   StemCells, Inc.
      2006   Phase 1   NCT00337636   United States
Melphalan
   Alice Bertaina
      2023   Phase 1/Phase 2   NCT05508009   United States
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
      2006   Phase 2   NCT00383448   United States
Mepsevii
   Ultragenyx Pharmaceutical Inc
      2017   -   NCT03775174   -
Mercaptamina
   Recordati Rare Diseases
      2020   Phase 3   EUCTR2018-002984-24-IT   Belgium;France;Germany;Italy;United Kingdom
Mercaptamine
   Recordati Rare Diseases
      2020   Phase 3   NCT04125927   Belgium;France;Germany;Italy;United Kingdom
Mercaptamine bitartrate
   HOSPICES CIVILS DE LYON
      2010   -   EUCTR2010-020098-18-FR   France
Mercaptamine hydrochloride
   Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR)
      2014   -   EUCTR2013-003228-35-ES   Spain
   Orphan Europe SARL
      2013   -   EUCTR2009-012564-13-FR   France
   RECORDATI Rare Diseases SARL
      2019   Phase 3   EUCTR2018-002984-24-GB   France;United Kingdom
Mercaptamine, mercamine, 2-aminoethanethiol
   Orphan Europe SARL
      2008   -   EUCTR2007-006024-35-FR   France
Metazym
   Shire Pharmaceutical Ireland Limited
      2008   -   EUCTR2007-006345-40-DK   Denmark
   Shire Pharmaceuticals Ireland Limited
      2008   Phase 2   EUCTR2007-007165-20-DK   Denmark
      2006   -   EUCTR2006-005341-11-DK   Denmark
Methotrexate
   Central Manchester University Hospitals NHS Foundation Trust
      2015   Phase 3   EUCTR2015-003031-35-GB   United Kingdom
   Takeda
      2023   Phase 4   NCT05494593   United States
Methylprednisolone
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
   Prevail Therapeutics
      2021   Phase 1/Phase 2   NCT04411654   United Kingdom;United States
Migalastat
   Amicus Therapeutics
      2020   -   NCT04252066   United States
      2011   Phase 1   NCT01489995   United States
   Manchester University NHS Foundation Trust
      2019   -   NCT03949920   United Kingdom
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
   Sanofi
      2022   Phase 3   NCT05280548   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
   Sanofi Aventis Groupe (SAG)
      -   Phase 4   EUCTR2019-000065-20-NO   Canada;France;Norway;United States
   Tanaka Tomoyuki
      2022   Phase 3   JPRN-jRCT2071220022   China;Italy;Japan;Republic of Korea
   sanofi-aventis recherche et développement
      2022   Phase 3   EUCTR2021-002320-20-PL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NO   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-DK   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-CZ   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-AT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002320-20-GR   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-002320-20-ES   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
Migalastat HCL
   Amicus Therapeutics
      2025   -   NCT06906367   -
      2011   Phase 2   NCT01196871   Australia;Belgium;Canada;France;Netherlands;United Kingdom;United States
      2007   Phase 2   NCT00526071   Australia;Brazil;France;United Kingdom;United States
      2006   Phase 2   NCT00304512   Australia;Brazil;Canada;France;United Kingdom;United States
      2006   Phase 2   NCT00283959   Australia;Brazil
      2006   Phase 2   NCT00283933   Canada;France;United Kingdom
      2006   Phase 2   NCT00214500   United States
   Koh KyuBum
      2023   Phase 3   JPRN-jRCT2051220125   Australia;France;Japan;Spain;USA
Migalastat HCL 150 MG
   Amicus Therapeutics
      2022   Phase 3   NCT04020055   Australia;Belgium;France;Italy;Japan;Portugal;Spain;United Kingdom;United States
      2019   Phase 3   NCT04049760   Spain;United Kingdom;United States
      2018   Phase 3   NCT03500094   Spain;United Kingdom;United States
      2014   Phase 3   NCT02194985   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2013   -   NCT01476163   Australia;United States
Migalastat HCL 20 MG
   Amicus Therapeutics
      2025   Phase 3   NCT06904261   United States
      2013   -   NCT01476163   Australia;United States
Migalastat hydrochloride
   AMICUS THERAPEUTICS, INC
      2010   -   EUCTR2009-013459-31-IT   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
   Amicus Therapeutics
      2011   Phase 3   NCT01458119   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2011   Phase 3   NCT01218659   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;Poland;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2009   Phase 3   NCT00925301   Argentina;Australia;Belgium;Brazil;Canada;Chile;Denmark;Egypt;France;Germany;Israel;Italy;Netherlands;Poland;South Africa;Spain;Turkey;United Kingdom;United States
      2005   Phase 2   EUCTR2005-004384-33-GB   United Kingdom
   Amicus Therapeutics UK, Ltd
      2020   Phase 3   EUCTR2017-000146-21-IT   Australia;Germany;Italy;Spain;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2015   Phase 3   EUCTR2014-002701-38-GB   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2015   Phase 3   EUCTR2014-002701-38-ES   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2015   Phase 3   EUCTR2014-002701-38-DK   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2015   Phase 3   EUCTR2014-002701-38-BE   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2014   Phase 3   EUCTR2014-002701-38-AT   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States
      2014   Phase 3   EUCTR2011-004800-40-AT   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2013   Phase 3   EUCTR2011-004800-40-BE   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2012   Phase 3   EUCTR2011-004800-40-GB   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2012   Phase 3   EUCTR2011-004800-40-DK   Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
      2012   -   EUCTR2010-022709-16-GB   Belgium;United Kingdom
      2012   -   EUCTR2010-022636-37-DE   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   Phase 2   EUCTR2010-022709-16-BE   Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom
      2011   Phase 3   EUCTR2010-022636-37-GR   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-GB   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-DK   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-BE   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-AT   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2010   Phase 3   EUCTR2009-013459-31-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2010   Phase 3   EUCTR2009-013459-31-ES   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2010   -   EUCTR2009-013459-31-DK   Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States
      2010   Phase 3   EUCTR2009-013459-31-BE   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 3   EUCTR2009-013459-31-GB   Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2009-013459-31-FR   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 2   EUCTR2007-001838-13-GB   Australia;Brazil;United Kingdom;United States
      2006   Phase 2   EUCTR2006-000181-36-GB   United Kingdom
      -   -   EUCTR2009-013459-31-DE   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
   Amicus Therapeutics, UK Limited
      2020   Phase 3   EUCTR2019-000222-21-GB   United Kingdom;United States
   Amicus Therapeutics, UK Ltd
      2019   Phase 3   EUCTR2017-000146-21-GB   Australia;Germany;Italy;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2017-000146-21-ES   Australia;Germany;Italy;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2017-000146-21-DE   Australia;Germany;Italy;Spain;United Kingdom;United States
   GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
      2012   -   EUCTR2011-004800-40-IT   Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States
   GlaxoSmithKline Research & Development Limited
      2012   Phase 3   EUCTR2011-004800-40-ES   Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States
Migalastat hydrocloride
   AMICUS THERAPEUTICS, INC
      2011   -   EUCTR2010-022636-37-IT   Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States
Miglustat
   Actelion
      2020   Phase 4   NCT03910621   China
      2006   Phase 3   NCT00319046   Australia;Brazil;Canada;Czech Republic;Czechia;France;Germany;Hungary;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
      2002   Phase 2   NCT00517153   United States
   Actelion Pharmaceuticals Ltd
      2007   -   EUCTR2005-001651-37-HU   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2006   -   EUCTR2005-001651-37-ES   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2006   -   EUCTR2005-001651-37-DE   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2006   -   EUCTR2005-001651-37-CZ   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2005   Phase 4   EUCTR2005-001651-37-GB   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
   Actelion Pharmaceuticals Trading (Shanghai) Co., Ltd
      -   Phase 4   EUCTR2022-002514-16-Outside-EU/EEA   China
   Actelion Registration Ltd
      2006   -   EUCTR2005-001651-37-IT   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
   Children's Research Institute
      2004   Phase 3   NCT00672022   United States
   Dipartimento di Pediatria Universita` di Napoli Federico II
      2006   -   EUCTR2006-005842-35-IT   Italy
   HOSPICES CIVILS DE LYON
      2006   -   EUCTR2006-004661-34-FR   France
   INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD
      2008   -   EUCTR2007-007164-20-ES   Spain
   ISTITUTO GIANNINA GASLINI
      2009   -   EUCTR2007-005516-61-IT   Italy
   National Taiwan University Hospital
      2015   -   NCT02520934   Taiwan
      2008   Phase 3   NCT01760564   Taiwan
   Orphazyme ApS
      2015   Phase 1   EUCTR2014-005194-37-ES   Germany;Spain;Switzerland
      2015   Phase 1   EUCTR2014-005194-37-DE   Germany;Spain;Switzerland
   Tehran University of Medical Sciences
      2019   Phase 3   NCT03822013   Iran, Islamic Republic of
   The Hospital for Sick Children
      2004   Phase 2   NCT00418847   Canada
   University of Minnesota
      2015   Phase 4   NCT02030015   United States
Montelukast
   Sanofi
      2023   Phase 4   NCT06019728   United States
MRI with contrast agent injection
   University Hospital, Bordeaux
      2016   -   NCT03123523   France
MRI without contrast agent injection
   University Hospital, Bordeaux
      2016   -   NCT03123523   France
Mucoangin
   Amsterdam UMC
      2022   Phase 3   EUCTR2021-002550-82-NL   Netherlands
Mycophenolate mofetil
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
      2009   Phase 2   NCT01043640   United States
   University of Rochester
      2011   Phase 2   NCT01399047   United States
Mycophenylate mofetil
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States
Myelostim - 34 1 flaconcino liofilizzato 33.6 MIU + siringa preriempita solvente 1 ML
   OSPEDALE SAN RAFFAELE
      2018   Phase 1   EUCTR2017-002430-23-IT   Italy
N acetyl cysteine
   University of Sao Paulo
      2011   Phase 4   NCT01614431   -
N acetyl L leucine
   IntraBio Ltd
      2022   Phase 3   EUCTR2021-005356-10-SK   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004406-25-ES   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-ES   Germany;Slovakia;Spain;United Kingdom;United States
N-acetyl-L leucine
   IntraBio Ltd
      2022   Phase 3   EUCTR2021-005356-10-NL   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-CZ   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States
N-acetyl-L-leucine
   IntraBio Inc
      2022   Phase 3   NCT05163288   Australia;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States
   IntraBio Ltd
      2022   Phase 3   EUCTR2021-005356-10-SK   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-NL   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-DE   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005356-10-CZ   Australia;Czech Republic;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004406-25-GB   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004406-25-ES   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004406-25-DE   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-SK   Germany;Slovakia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-GB   Germany;Slovakia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-DE   Germany;Slovakia;Spain;United Kingdom;United States
N-acetylcysteine
   University of Minnesota
      2015   Phase 2   NCT02583672   United States
      2011   Phase 1   NCT01427517   United States
N-acetylcysteine amide
   Nacuity Pharmaceuticals, Inc.
      2023   Phase 1/Phase 2   NCT05994534   Australia
N-acetylgalactosamine 4-sulfatase
   BioMarin Pharmaceutical
      2004   Phase 3   NCT00104234   United States
      2003   Phase 3   NCT00067470   United States
      2002   Phase 2   NCT00048711   United States
      2000   Phase 1   NCT00048620   United States
Naglazyme
      2007   -   EUCTR2007-001453-26-NL   Netherlands
   BioMarin Pharmaceutical
      2006   Phase 4   NCT00299000   France;Portugal;United States
   BioMarin Pharmaceutical Inc
      2007   -   EUCTR2005-003512-30-PT   France;Portugal
   BioMarin Pharmaceutical Inc.
      2007   Phase 4   EUCTR2005-003512-30-FR   France;Portugal
   Masonic Cancer Center, University of Minnesota
      2016   -   NCT02156674   United States
Neurocognitive testing
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Neurology exam
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
NGN-101
   Neurogene Inc.
      2022   Phase 1/Phase 2   NCT05228145   United Kingdom;United States
NO investigational product
   Ultragenyx Pharmaceutical Inc
      2020   Phase 3   NCT04360265   Australia;Spain;United States
NO IT treatment
   Shire
      2014   Phase 2/Phase 3   NCT02055118   Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States
NON applicable
   Inserm
      2012   Phase 1;Phase 2   EUCTR2011-004410-42-FR   France
Odiparcil
   Inventiva Pharma
      2017   Phase 2   NCT03370653   France;Germany;Portugal;United Kingdom
   Inventiva S.A.
      2019   Phase 2   EUCTR2017-002158-35-PT   France;Germany;Portugal;United Kingdom
      2018   Phase 2   EUCTR2017-002158-35-DE   France;Germany;Portugal;United Kingdom
      2017   Phase 2   EUCTR2017-002158-35-GB   France;Germany;Portugal;United Kingdom
      -   Phase 2   EUCTR2017-002158-35-FR   France;Germany;Portugal;United Kingdom
OGT 918
   National Institute of Neurological Disorders and Stroke (NINDS)
      2002   Phase 2   NCT00041535   United Kingdom;United States
OGT918
   Actelion Pharmaceuticals Ltd
      2007   -   EUCTR2005-001651-37-HU   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2006   -   EUCTR2005-001651-37-ES   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2006   -   EUCTR2005-001651-37-DE   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2006   -   EUCTR2005-001651-37-CZ   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2005   Phase 4   EUCTR2005-001651-37-GB   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
   National Eye Institute (NEI)
      2002   Phase 1   NCT00316498   United Kingdom;United States
OGT923
   IDORSIA PHARMACEUTICALS LTD
      2020   Phase 3   EUCTR2018-002210-12-IT   Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-IT   Australia;Austria;Belgium;Canada;Finland;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
   Idorsia Pharmaceuticals Ltd
      2021   Phase 3   EUCTR2018-002210-12-NO   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2021   Phase 3   EUCTR2018-002210-12-FR   Austria;Belgium;Canada;Czech Republic;France;Germany;Netherlands;Poland;United Kingdom;United States
      2020   Phase 3   EUCTR2018-002210-12-BE   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-NO   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-IE   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2020   Phase 3   EUCTR2017-003369-85-ES   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Russian Federation;Spain;Switzerland;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-NL   Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-GB   Australia;Austria;Belgium;Canada;Germany;Ireland;Netherlands;Poland;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-DE   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-002210-12-AT   Australia;Austria;Belgium;Canada;France;Germany;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2017-003369-85-NL   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2018   Phase 3   EUCTR2017-003369-85-GB   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
      2018   Phase 3   EUCTR2017-003369-85-AT   Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States
Olenasufligene relduparvovec
   Lysogene SA
      2019   Phase 2;Phase 3   EUCTR2018-000195-15-NL   France;Germany;Netherlands;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-000195-15-GB   France;Germany;Netherlands;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-000195-15-DE   France;Germany;Netherlands;United Kingdom;United States
      2018   Phase 2;Phase 3   EUCTR2018-000195-15-FR   France;Germany;Netherlands;United Kingdom;United States
Olipudase alfa
   GENZYME CORPORATION
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-IT   Argentina;Australia;Brazil;Chile;France;Germany;Israel;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
   Genzyme Corporation
      2019   Phase 2   EUCTR2013-000051-40-BE   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2018   Phase 2   EUCTR2013-000051-40-DE   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-PT   Argentina;Australia;Belgium;Brazil;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-NL   Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2016   Phase 2   EUCTR2013-000051-40-IT   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2;Phase 3   EUCTR2015-000371-26-GB   Argentina;Australia;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   -   EUCTR2015-000371-26-ES   Argentina;Australia;Brazil;Chile;France;Germany;Israel;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   Phase 2;Phase 3   EUCTR2015-000371-26-DE   Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-GB   Brazil;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-DE   Brazil;France;Germany;Italy;United Kingdom;United States
      2013   Phase 2   EUCTR2013-000051-40-GB   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
   Genzyme, a Sanofi Company
      2015   Phase 1/Phase 2   NCT02292654   Brazil;France;Germany;Italy;United Kingdom;United States
   Sanofi
      2024   -   NCT06192576   Canada;United States
      2022   -   NCT05359276   France
      2021   Phase 2   NCT06949358   France
      2021   -   NCT04877132   -
   Tanaka Tomoyuki
      2021   Phase 2-3   JPRN-jRCT2031210279   -
Omnipaque
   Genzyme, a Sanofi Company
      2012   Phase 1   EUCTR2012-001966-14-ES   Canada;Netherlands;Norway;Spain;United Kingdom;United States
Ophthalmology exam
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Optimized prophylactic IM therapy
   Ultragenyx Pharmaceutical Inc
      2016   Phase 2/Phase 3   NCT02716246   Australia;France;Germany;Spain;United States
Oral migalastat HCL
   Amicus Therapeutics
      2014   Phase 1   NCT02082327   Netherlands
Osome
   Université de Montréal
      2014   -   NCT02023086   Canada
Osteopetrosis haploidentical only preparative regimen
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States
Osteopetrosis only preparative regimen
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States
OTL-200
   Orchard Therapeutics
      2022   Phase 3   NCT04283227   Italy
      2018   Phase 2   NCT03392987   Italy
OTL-200 dispersion FOR infusion
   Orchard Therapeutics (Europe) Limited
      2019   Phase 3   EUCTR2019-002636-82-IT   Italy
   Orchard Therapeutics (Europe) Ltd
      2017   Phase 3   EUCTR2017-001730-26-IT   Italy
OTL-200 gene therapy
   Orchard Therapeutics
      2010   Phase 1/Phase 2   NCT01560182   Italy
OTL-203
   Orchard Therapeutics
      2023   Phase 3   NCT06149403   Italy;Netherlands;United Kingdom;United States
Oxygen flow AT THE optic nerve head measurement
   Université de Montréal
      2014   -   NCT02023086   Canada
Pabinafusp alfa
   Ibaraki Ryo
      2021   Phase 4   JPRN-jRCT2031210126   -
   JCR Pharmaceutical Co., Ltd.
      2023   Phase 3   EUCTR2020-003200-14-IT   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Turkey;United Kingdom;United States
   JCR Pharmaceuticals Co., Ltd.
      2023   Phase 3   EUCTR2020-003200-14-PL   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003200-14-FR   Brazil;France;Germany;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003200-14-DE   Argentina;Brazil;Colombia;France;Germany;Italy;Poland;Spain;Türkiye;United Kingdom;United States
Palliative care
   University of Pittsburgh
      2012   -   NCT03333200   United States
Paracetamolo DOC generici - 1000 MG compressa 16 compresse
   Sangamo Therapeutics, Inc.
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-IT   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
Paricalcitol
   Federico II University
      2012   -   NCT02090608   Italy
PBGM01
   Gemma Biotherapeutics
      2021   Phase 1/Phase 2   NCT04713475   Brazil;Canada;Turkey;United Kingdom;United States
PBKR03
   Passage Bio, Inc.
      2022   Phase 1/Phase 2   NCT04771416   Brazil;Canada;Israel;Netherlands;United Kingdom;United States
      2021   Phase 1;Phase 2   EUCTR2020-005229-95-NL   Brazil;Canada;Israel;Netherlands;United Kingdom;United States
Pegunigalsidase alfa
   Chiesi Farmaceutici S.p.A
      2021   Phase 3   EUCTR2018-001148-67-FI   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2018-001148-67-SI   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001947-30-DK   Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001148-67-NL   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001148-67-HU   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001148-67-NO   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001148-67-CZ   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
   Chiesi Farmaceutici S.p.A.
      2021   -   NCT04552691   United States
      2019   Phase 3   EUCTR2018-001947-30-BE   Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States
      2018   Phase 3   NCT03614234   Belgium;Czechia;Denmark;Italy;Norway;Spain;United Kingdom;United States
      2018   Phase 3   NCT03566017   Australia;Canada;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
   PROTALIX LTD
      2020   Phase 3   EUCTR2018-001148-67-IT   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001947-30-IT   Australia;Belgium;Canada;Czechia;Denmark;Finland;France;Italy;Netherlands;Norway;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-001528-23-IT   Austria;Belgium;Canada;Czech Republic;Czechia;Denmark;Germany;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000378-38-IT   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
   Protalix
      2017   Phase 3   NCT03180840   Belgium;Canada;Czechia;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 3   NCT03018730   Australia;Canada;Czech Republic;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2016   Phase 3   NCT02795676   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2014   Phase 1/Phase 2   NCT01981720   Paraguay;Spain;United Kingdom;United States
   Protalix Ltd.
      2021   Phase 3   EUCTR2018-001148-67-FR   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001947-30-GB   Australia;Belgium;Canada;Czech Republic;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001148-67-ES   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2016-000378-38-FR   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2016-000378-38-FI   Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001148-67-GB   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-001528-23-DK   Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-001528-23-CZ   Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-001318-11-NL   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2017-001528-23-GB   Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2017-001528-23-ES   Belgium;Canada;Czech Republic;Denmark;Germany;Italy;Netherlands;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2017-001528-23-BE   Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-001318-11-SI   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-GB   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-ES   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-DE   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-CZ   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-000378-38-SI   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000378-38-NL   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-NO   Australia;Belgium;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-HU   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-GB   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-ES   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-BE   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
Pegunigalsidase alpha
   PROTALIX LTD
      2017   Phase 3   EUCTR2016-000378-38-IT   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
   Protalix Ltd.
      2019   Phase 3   EUCTR2016-000378-38-FR   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2016-000378-38-FI   Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-001318-11-NL   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-SI   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-GB   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-ES   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-DE   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-CZ   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-000378-38-SI   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000378-38-NL   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-NO   Australia;Belgium;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-HU   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-GB   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-ES   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-BE   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
Pegunigalsidase-alfa
   Chiesi Farmaceutici S.p.A.
      2024   -   NCT06663358   United States
   Universität Münster
      2023   -   NCT06095713   Germany
Pending
   Ultragenyx Pharmaceutical Inc.
      2016   Phase 3   EUCTR2015-001875-32-PT   Brazil;Mexico;Portugal;United States
      -   -   EUCTR2015-001875-32-Outside-EU/EEA   United States
      -   -   EUCTR2015-000104-26-Outside-EU/EEA   United States
Pentosan polysulfat SP 54® injection solution
   Multiplex Pharma Holdings LLC
      2014   -   EUCTR2014-000350-11-DE   Germany
Pentosan polysulfate sodium
   Multiplex Pharma Holdings LLC
      2014   -   EUCTR2014-000350-11-DE   Germany
Pentosan polysulphate SP54
   Multiplex Pharma Holdings LLC
      2014   -   EUCTR2014-000350-11-DE   Germany
Perdix
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Picc or other central line placement
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Plant cell recombinant glucocerebrosidase
   PROTALIX BIOTHERAPEUTICS
      2009   -   EUCTR2008-005826-35-IT   Italy;Spain;United Kingdom
      2009   -   EUCTR2008-005825-12-IT   Germany;Italy;Spain;United Kingdom
      2008   -   EUCTR2007-000498-42-IT   Italy;Spain;United Kingdom
PLX-200
   Polaryx Therapeutics, Inc.
      2024   Phase 3   NCT04637282   -
Potable water
   Amicus Therapeutics
      2011   Phase 1   NCT01853852   Australia
Prednisone
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
   Prevail Therapeutics
      2021   Phase 1/Phase 2   NCT04411654   United Kingdom;United States
Prednisone DOC generici - 5 MG compresse 10 compresse IN blister PVC-pvdc/ALU
   Sangamo Therapeutics, Inc.
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-IT   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
Prevenar13
   Maastricht University
      2016   Phase 4   EUCTR2015-004846-25-BE   Belgium
Prgcd
   PROTALIX BIOTHERAPEUTICS
      2009   -   EUCTR2008-005826-35-IT   Italy;Spain;United Kingdom
      2009   -   EUCTR2008-005825-12-IT   Germany;Italy;Spain;United Kingdom
      2008   -   EUCTR2007-000498-42-IT   Italy;Spain;United Kingdom
   Pfizer
      2007   Phase 3   NCT00376168   Canada;Chile;Israel;Italy;South Africa;Spain;United Kingdom;United States
      2006   -   NCT00962260   Israel;United States
   Protalix
      2005   Phase 1   NCT00258778   Israel
   Protalix Biotherapeutics
      2012   Phase 3   EUCTR2011-003685-32-ES   Canada;Chile;Israel;Italy;Mexico;Serbia;South Africa;Spain
      2011   Phase 3   EUCTR2011-003676-37-ES   Israel;South Africa;Spain
      2009   Phase 3   EUCTR2008-005826-35-GB   Italy;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005826-35-ES   Italy;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005825-12-GB   Germany;Italy;Spain;United Kingdom
      2009   -   EUCTR2008-005825-12-ES   Germany;Italy;Spain;United Kingdom
      2009   -   EUCTR2008-005825-12-DE   Germany;Italy;Spain;United Kingdom
      2008   Phase 3   EUCTR2007-000498-42-GB   Italy;Spain;United Kingdom
      2008   -   EUCTR2007-000498-42-ES   Italy;Spain;United Kingdom
      -   -   EUCTR2008-005826-35-Outside-EU/EEA   Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States
      -   -   EUCTR2008-005825-12-Outside-EU/EEA   Canada;Israel;United Kingdom;United States
      -   -   EUCTR2007-000498-42-Outside-EU/EEA   Israel;United States
Procysbi
   Horizon Pharma USA, Inc.
      2014   Phase 3   EUCTR2012-002773-64-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2013   Phase 3   EUCTR2012-002773-64-NL   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2010   Phase 3   EUCTR2010-018365-34-NL   France;Netherlands;United States
Prophylactic immunomodulatory (IM) therapy
   Ultragenyx Pharmaceutical Inc
      2016   Phase 2/Phase 3   NCT02716246   Australia;France;Germany;Spain;United States
PRX-102
   Chiesi Farmaceutici S.p.A
      2021   Phase 3   EUCTR2018-001148-67-FI   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2018-001148-67-SI   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001947-30-DK   Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001148-67-NL   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001148-67-HU   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001148-67-NO   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001148-67-CZ   Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States
   Chiesi Farmaceutici S.p.A.
      2019   Phase 3   EUCTR2018-001947-30-BE   Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States
   PROTALIX LTD
      2020   Phase 3   EUCTR2018-001148-67-IT   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001947-30-IT   Australia;Belgium;Canada;Czechia;Denmark;Finland;France;Italy;Netherlands;Norway;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-001528-23-IT   Austria;Belgium;Canada;Czech Republic;Czechia;Denmark;Germany;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000378-38-IT   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
   Protalix
      2017   Phase 3   NCT03018730   Australia;Canada;Czech Republic;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   NCT02921620   -
      2016   Phase 3   NCT02795676   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2014   Phase 1/Phase 2   NCT01981720   Paraguay;Spain;United Kingdom;United States
      2013   Phase 1/Phase 2   NCT01769001   Australia;Paraguay;Spain;United Kingdom;United States
      2012   Phase 1/Phase 2   NCT01678898   Australia;Paraguay;Serbia;Spain;United Kingdom;United States
   Protalix Ltd.
      2021   Phase 3   EUCTR2018-001148-67-FR   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001947-30-GB   Australia;Belgium;Canada;Czech Republic;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001148-67-ES   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2016-000378-38-FR   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2019   Phase 3   EUCTR2016-000378-38-FI   Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001148-67-GB   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-001528-23-DK   Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2017-001528-23-CZ   Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-001318-11-NL   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2017-001528-23-GB   Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2017-001528-23-ES   Belgium;Canada;Czech Republic;Denmark;Germany;Italy;Netherlands;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2017-001528-23-BE   Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-001318-11-SI   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-GB   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-ES   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-DE   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-001318-11-CZ   Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom
      2017   Phase 3   EUCTR2016-000378-38-SI   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000378-38-NL   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-NO   Australia;Belgium;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-HU   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-GB   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-ES   Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000378-38-BE   Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States
      2015   Phase 1;Phase 2   EUCTR2014-005544-18-GB   Paraguay;Spain;United Kingdom;United States
      2013   Phase 1;Phase 2   EUCTR2013-002554-78-GB   Argentina;Australia;Chile;Israel;Paraguay;Spain;United Kingdom;United States
      2013   -   EUCTR2012-004786-40-GB   Argentina;Australia;Chile;Israel;Paraguay;Spain;United Kingdom;United States
PRX-102 1 MG/KG every 2 weeks
   Chiesi Farmaceutici S.p.A.
      2023   Phase 2/Phase 3   NCT05710692   Japan
PRX-102 1 MG/KG every TWO weeks
   Chiesi Farmaceutici S.p.A.
      2025   Phase 2/Phase 3   NCT06328608   Austria;France;Norway;Spain;United Kingdom;United States
PRX-102 2 MG/KG every 4 weeks
   Chiesi Farmaceutici S.p.A.
      2023   Phase 2/Phase 3   NCT05710692   Japan
PRX-112
   Protalix
      2014   Phase 2   NCT02107846   Israel
      2013   Phase 1   NCT01747980   Israel
PRX102
   Protalix Ltd.
      2015   Phase 1;Phase 2   EUCTR2014-005544-18-ES   Paraguay;Spain;United Kingdom;United States
      2013   Phase 1;Phase 2   EUCTR2013-002554-78-ES   Australia;Israel;Paraguay;Serbia;Spain;United Kingdom;United States
      2013   -   EUCTR2012-004786-40-ES   Argentina;Australia;Israel;Paraguay;Serbia;Spain;United Kingdom;United States
PTC124
   PTC Therapeutics Inc.
      2015   Phase 2   EUCTR2015-003105-41-GB   United Kingdom
      2015   Phase 2   EUCTR2014-002596-28-GB   Germany;United Kingdom
Purified form OF THE lysosomal enzyme iduronate-2-sulfatase
   Denali Therapeutics Inc.
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-SE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-NL   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-IT   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-FR   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-CZ   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-BE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-005200-35-DE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
Pyrimethamine
   Exsar Corporation
      2008   Phase 1   NCT00679744   Canada;United States
   The Hospital for Sick Children
      2009   Phase 1/Phase 2   NCT01102686   Canada
Quality OF life questionnaire
   Baylor Research Institute
      2018   -   NCT04002531   United States
Raav 2/6 hids donor
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000192-33-GB   United Kingdom;United States
RAAV2/5-hnaglu
   Institut Pasteur
      -   Phase 1;Phase 2   EUCTR2012-000856-33-FR   France
   UniQure Biopharma B.V.
      2013   Phase 1/Phase 2   NCT03300453   France
RAAV2/6 left ZFN vector
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000206-28-GB   United Kingdom;United States
      2018   Phase 1;Phase 2   EUCTR2018-000192-33-GB   United Kingdom;United States
RAAV2/6 right ZFN vector
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000206-28-GB   United Kingdom;United States
      2018   Phase 1;Phase 2   EUCTR2018-000192-33-GB   United Kingdom;United States
RAAV9.CMV.hnaglu
   Abeona Therapeutics Europe SL.
      2021   Phase 1;Phase 2   EUCTR2019-002936-97-FR   France;Germany;United States
      2019   Phase 1;Phase 2   EUCTR2014-001411-39-GB   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2019   Phase 1;Phase 2   EUCTR2014-001411-39-DE   France;Germany;Spain;United Kingdom;United States
      -   Phase 1;Phase 2   EUCTR2019-002936-97-DE   France;Germany;Spain;United States
      -   Phase 1;Phase 2   EUCTR2014-001411-39-FR   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Abeona Therapeutics Inc
      2018   Phase 1;Phase 2   EUCTR2014-001411-39-ES   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
Rebisufligene etisparvovec
   Abeona Therapeutics Inc
      2020   Phase 1;Phase 2   EUCTR2019-002979-34-ES   Australia;France;Germany;Spain;United States
      2019   Phase 1;Phase 2   EUCTR2015-003904-21-FR   Australia;Brazil;France;Germany;Spain;United Kingdom;United States
Recombinant adeno-associated virus 2/6 vector encoding THE cdna FOR human alpha galactosidase A
   Sangamo Therapeutics, Inc.
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-IT   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-DE   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
      2019   Phase 1;Phase 2   EUCTR2019-000667-24-GB   United Kingdom;United States
Recombinant alpha-galactosidase A
   Assistance Publique - Hôpitaux de Paris
      2005   Phase 4   NCT00487630   France
Recombinant human acid sphingomyelinase
   Genzyme Corporation
      2019   Phase 2   EUCTR2013-000051-40-BE   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2018   Phase 2   EUCTR2013-000051-40-DE   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-PT   Argentina;Australia;Belgium;Brazil;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-NL   Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2016   Phase 2   EUCTR2013-000051-40-IT   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2;Phase 3   EUCTR2015-000371-26-GB   Argentina;Australia;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   -   EUCTR2015-000371-26-ES   Argentina;Australia;Brazil;Chile;France;Germany;Israel;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   Phase 2;Phase 3   EUCTR2015-000371-26-DE   Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-IT   Brazil;Chile;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-GB   Brazil;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-DE   Brazil;France;Germany;Italy;United Kingdom;United States
      2014   -   EUCTR2010-023953-12-IT   Brazil;Chile;France;Germany;Italy;Tunisia;United Kingdom;United States
      2013   Phase 2   EUCTR2013-000051-40-GB   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2013   -   EUCTR2010-023953-12-GB   Brazil;Chile;France;Germany;Italy;Tunisia;United Kingdom;United States
      2013   -   EUCTR2010-023953-12-FR   Brazil;Chile;France;Germany;Italy;Tunisia;United Kingdom;United States
      -   Phase 2   EUCTR2014-003198-40-FR   Brazil;Chile;France;Germany;Italy;United Kingdom;United States
   Genzyme, a Sanofi Company
      2013   Phase 1   NCT01722526   United Kingdom;United States
Recombinant human alpha-galactosidase
   Bio Sidus SA
      2022   Phase 3   NCT05843916   Argentina
   Genzyme Europe BV
      2008   -   EUCTR2007-005668-28-GB   Argentina;Brazil;Canada;Czech Republic;France;Germany;Netherlands;Norway;Poland;Portugal;United Kingdom;United States
   Greenovation Biotech GmbH
      2016   Phase 1   NCT02995993   Germany
Recombinant human alpha-galactosidase abbreviated AS R-H-alpha-GAL
   Genzyme Europe BV
      2009   Phase 3   EUCTR2007-005668-28-FR   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-PT   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-NL   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-DE   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
      2008   -   EUCTR2007-005668-28-CZ   Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom
Recombinant human alpha-mannosidase
   CHIESI FARMACEUTICI S.P.A.
      2018   Phase 2   EUCTR2016-001988-36-IT   Austria;Denmark;France;Germany;Italy
   Chiesi Farmaceutici S.p.A.
      2017   Phase 2   EUCTR2016-001988-36-FR   Austria;Denmark;France;Germany;Italy
      2017   Phase 2   EUCTR2016-001988-36-DE   Austria;Denmark;Germany
      2017   Phase 2   EUCTR2016-001988-36-AT   Austria;Denmark;Germany
      2016   Phase 2   EUCTR2016-001988-36-DK   Austria;Denmark;Germany;Italy
      2013   Phase 3   EUCTR2013-000321-31-DK   Denmark;Poland
      -   Phase 3   EUCTR2013-000321-31-PL   Denmark;Poland
   Zymenex A/S
      2013   -   EUCTR2012-000979-17-SE   Belgium;Denmark;Germany;Spain;Sweden
      2013   Phase 3   EUCTR2012-000979-17-ES   Belgium;Denmark;Germany;Spain;Sweden
      2013   -   EUCTR2012-000979-17-BE   Belgium;Denmark;Germany;Spain;Sweden
      2012   -   EUCTR2012-000979-17-DK   Belgium;Denmark;Germany;Spain;Sweden
      2012   -   EUCTR2012-000979-17-DE   Belgium;Denmark;Germany;Spain;Sweden
      2012   -   EUCTR2011-004355-40-GB   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   Phase 2   EUCTR2011-004355-40-DK   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   -   EUCTR2011-004355-40-DE   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   -   EUCTR2011-004355-40-BE   Belgium;Denmark;Germany;Spain;United Kingdom
      2010   -   EUCTR2010-022085-26-DK   Denmark
      2010   -   EUCTR2010-022084-36-DK   Denmark
Recombinant human arylsulfatase A
   SHIRE HUMAN GENETIC THERAPIES, INC
      2020   Phase 2   EUCTR2018-003291-12-IT   Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States
   Shire
      2012   Phase 1/Phase 2   NCT01510028   Argentina;Australia;Brazil;Denmark;France;Germany;Japan
      2007   Phase 1/Phase 2   NCT00633139   Denmark
   Shire Human Genetic Therapies Inc
      2010   Phase 2   EUCTR2008-000084-41-BE   Belgium;Denmark;France;Italy
      2009   Phase 2   EUCTR2008-000084-41-FR   Belgium;Denmark;France;Italy
      2008   Phase 2   EUCTR2008-000084-41-DK   Belgium;Denmark;France;Italy
   Shire Human Genetic Therapies, Inc.
      2021   Phase 2   EUCTR2018-003291-12-GR   Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States
      2020   Phase 2   EUCTR2018-003291-12-DE   Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-NL   Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-GB   Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-FR   Belgium;Brazil;Canada;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-ES   Belgium;Brazil;Canada;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-BE   Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetics Therapies Inc
      2017   Phase 1;Phase 2   EUCTR2012-003775-20-IT   Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom
      2016   Phase 1;Phase 2   EUCTR2012-003775-20-GB   Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom
      2014   Phase 1;Phase 2   EUCTR2012-003775-20-CZ   Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom
      2012   Phase 1;Phase 2   EUCTR2012-003775-20-FR   Argentina;Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;United Kingdom
      2012   Phase 1;Phase 2   EUCTR2011-002044-28-DE   Australia;Brazil;Denmark;France;Germany;Japan
      2011   Phase 1;Phase 2   EUCTR2011-002044-28-DK   Australia;Brazil;Denmark;France;Germany;Japan
   Shire Pharmaceutical Ireland Limited
      2008   -   EUCTR2007-006345-40-DK   Denmark
   Shire Pharmaceuticals Ireland Limited
      2008   Phase 2   EUCTR2007-007165-20-DK   Denmark
Recombinant human arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase
   BioMarin Pharmaceutical Inc.
      2007   Phase 4   EUCTR2005-003512-30-FR   France;Portugal
Recombinant human beta glucuronidase
   Ultragenyx Pharmaceutical Inc.
      2016   Phase 3   EUCTR2015-001875-32-PT   Brazil;Mexico;Portugal;United States
      2016   Phase 2   EUCTR2015-000104-26-PT   Portugal;Spain;United States
      2016   Phase 2   EUCTR2015-000104-26-ES   Portugal;Spain;United States
      2014   Phase 1;Phase 2   EUCTR2013-001152-35-ES   Spain;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2013-001152-35-GB   Spain;United Kingdom
      -   -   EUCTR2015-001875-32-Outside-EU/EEA   United States
      -   -   EUCTR2015-000104-26-Outside-EU/EEA   United States
      -   -   EUCTR2014-005638-71-Outside-EU/EEA   United States
Recombinant human BETA-glucuronidase
   Hamazaki Takashi
      2020   Phase 3   JPRN-jRCT2051190085   -
   Ultragenyx Pharmaceutical Inc.
      2016   Phase 3   EUCTR2015-001875-32-PT   Brazil;Mexico;Portugal;United States
      2016   Phase 2   EUCTR2015-000104-26-PT   Portugal;Spain;United States
      2016   Phase 2   EUCTR2015-000104-26-ES   Portugal;Spain;United States
      2014   Phase 1;Phase 2   EUCTR2013-001152-35-ES   Spain;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2013-001152-35-GB   Spain;United Kingdom
      -   -   EUCTR2015-001875-32-Outside-EU/EEA   United States
      -   -   EUCTR2014-005638-71-Outside-EU/EEA   United States
Recombinant human betaglucuronidase
   Ultragenyx Pharmaceutical Inc.
      -   -   EUCTR2015-000104-26-Outside-EU/EEA   United States
Recombinant human derived macrophage-targeted ß-glucocerebrosidase
   Genzyme Europe B.V.
      2010   -   EUCTR2008-005223-28-DE   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2008-005223-28-NL   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005223-28-GB   Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2009   Phase 3   EUCTR2008-005223-28-FR   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2009   -   EUCTR2008-005223-28-CZ   Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom
Recombinant human glucerebrosidase
   Protalix Biotherapeutics
      2012   Phase 3   EUCTR2011-003685-32-ES   Canada;Chile;Israel;Italy;Mexico;Serbia;South Africa;Spain
      2011   Phase 3   EUCTR2011-003676-37-ES   Israel;South Africa;Spain
Recombinant human glucocerebrosidase
   Protalix Biotherapeutics
      2009   Phase 3   EUCTR2008-005826-35-GB   Italy;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005826-35-ES   Italy;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005825-12-GB   Germany;Italy;Spain;United Kingdom
      2009   -   EUCTR2008-005825-12-ES   Germany;Italy;Spain;United Kingdom
      2009   -   EUCTR2008-005825-12-DE   Germany;Italy;Spain;United Kingdom
      2008   Phase 3   EUCTR2007-000498-42-GB   Italy;Spain;United Kingdom
      2008   -   EUCTR2007-000498-42-ES   Italy;Spain;United Kingdom
Recombinant human heparan N-sulfatase
   SHIRE HUMAN GENETIC THERAPIES, INC
      2015   Phase 2   EUCTR2014-003960-20-IT   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire
      2014   Phase 2   NCT02060526   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2010   Phase 1/Phase 2   NCT01155778   Netherlands;United Kingdom
   Shire Human Genetic Therapies, Inc
      2014   Phase 2   EUCTR2013-003450-24-NL   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003450-24-IT   France;Germany;Italy;Netherlands;Spain;United Kingdom
      2014   Phase 2   EUCTR2013-003450-24-GB   Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003450-24-DE   Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2011   Phase 1;Phase 2   EUCTR2010-021348-16-NL   Netherlands;United Kingdom
      2011   Phase 1;Phase 2   EUCTR2010-021348-16-GB   Netherlands;United Kingdom
      -   Phase 2   EUCTR2013-003450-24-FR   Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetic Therapies, Inc.
      2015   Phase 2   EUCTR2014-003960-20-NL   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003960-20-GB   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003960-20-DE   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetic Therapies, Inc. (Shire HGT)
      2010   Phase 1;Phase 2   EUCTR2009-015984-15-GB   United Kingdom
   Shire human Genetic Therapies, Inc.
      2015   Phase 2   EUCTR2014-003960-20-ES   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      -   Phase 2   EUCTR2014-003960-20-FR   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
Recombinant human lysosomal acid lipase
   SYNAGEVA BIOPHARMA CORP.
      2012   -   EUCTR2011-000032-28-IT   Germany;Ireland;Italy;Taiwan;United Kingdom;United States
   Synageva Biopharma Corp.
      2011   -   EUCTR2011-002880-42-GB   France;Germany;United Kingdom;United States
      2011   Phase 2   EUCTR2011-001513-13-GB   Czech Republic;France;United Kingdom;United States
      2011   Phase 2   EUCTR2011-001513-13-CZ   Czech Republic;France;United Kingdom;United States
      2011   Phase 1;Phase 2;Phase 3   EUCTR2011-000032-28-GB   France;Germany;Ireland;Italy;Taiwan;United Kingdom;United States
      2011   -   EUCTR2010-024068-16-GB   Czech Republic;United Kingdom
      2011   -   EUCTR2010-024068-16-CZ   Czech Republic;United Kingdom;United States
      -   Phase 2   EUCTR2011-002880-42-FR   France;Germany;United Kingdom;United States
      -   Phase 1;Phase 2   EUCTR2011-000032-28-FR   France;Germany;Ireland;Italy;United Kingdom;United States
Recombinant human lysosomal alpha-mannosidase
   Zymenex A/S
      2015   -   EUCTR2014-003950-15-DK   Denmark
      2013   Phase 3   EUCTR2013-000336-97-FR   Denmark;France
Recombinant human N-acetylgalactosamine 4-sulfatase
   BioMarin Pharmaceutical Inc.
      -   Phase 3   EUCTR2004-000642-21-IE   Ireland
Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110
   BIOMARIN PHARMACEUTICAL INC.
      2011   -   EUCTR2010-020199-45-IT   Argentina;Australia;Brazil;Canada;Colombia;Denmark;Germany;Italy;Japan;Korea, Democratic People's Republic of;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States
   BioMarin Pharmaceutical Inc.
      2012   -   EUCTR2010-020199-45-PT   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2012   Phase 3   EUCTR2010-020199-45-DE   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States
Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110
   BioMarin Pharmaceutical Inc.
      2013   Phase 3   EUCTR2010-020199-45-ES   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States
      2012   Phase 3   EUCTR2010-020199-45-DK   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States
Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
   BIOMARIN PHARMACEUTICAL INC.
      2012   Phase 2   EUCTR2011-003197-84-IT   Italy;United Kingdom;United States
      2011   -   EUCTR2010-020198-18-IT   Denmark;Germany;Italy;Norway;Portugal;United Kingdom
   BioMarin Pharmaceutical Inc
      2012   Phase 3   EUCTR2010-020198-18-NO   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States
      2011   -   EUCTR2010-020198-18-PT   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States
      2011   -   EUCTR2010-020198-18-DK   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States
      2011   -   EUCTR2010-020198-18-DE   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States
      2010   -   EUCTR2010-020198-18-GB   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2010-020198-18-NL   Denmark;France;Germany;Italy;Netherlands;Norway;Portugal;United Kingdom
      -   Phase 3   EUCTR2010-020198-18-FR   Denmark;France;Germany;Italy;Netherlands;Norway;Portugal;United Kingdom
   BioMarin Pharmaceutical Inc.
      2012   -   EUCTR2011-005703-33-GB   Germany;Italy;United Kingdom;United States
      2012   -   EUCTR2011-005703-33-DE   Germany;Italy;United Kingdom;United States
      2012   -   EUCTR2011-005682-20-GB   Canada;Germany;United Kingdom;United States
      2012   -   EUCTR2011-005682-20-DE   Canada;Germany;United Kingdom;United States
      2011   Phase 2   EUCTR2011-003197-84-GB   Italy;United Kingdom;United States
      2011   -   EUCTR2010-020199-45-GB   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;United Kingdom;United States
      2010   -   EUCTR2010-021048-16-GB   United Kingdom
Recombinant human tripeptidyl peptidase-1
   BioMarin Pharmaceutical
      2016   Phase 2   NCT02678689   Germany;Italy;United Kingdom;United States
   BioMarin Pharmaceutical Inc
      2017   Phase 2   EUCTR2015-000891-85-GB   Germany;Italy;United Kingdom;United States
   BioMarin Pharmaceutical Inc.
      2016   Phase 2   EUCTR2015-000891-85-DE   Germany;Italy;United Kingdom
      2015   -   EUCTR2014-003480-37-IT   Germany;Italy;Japan;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003480-37-GB   Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003480-37-DE   Germany;Italy;United Kingdom;United States
      2014   Phase 1;Phase 2   EUCTR2012-005430-11-IT   Germany;Italy;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2012-005430-11-GB   Germany;Italy;United Kingdom;United States
      2013   Phase 1;Phase 2   EUCTR2012-005430-11-DE   Germany;United Kingdom
Recombinant SELF-complementary adeno-associated viral vector serotype 9 containing THE human CLN6 gene
   Amicus Therapeutics, Inc.
      2021   Phase 1;Phase 2   EUCTR2021-000706-21-DE   Germany;United States
Reduced intensity conditioning
   Duke University
      2008   Phase 1   NCT00744692   United States
Reminder capsules
   Amicus Therapeutics, Inc.
      2009   Phase 2   EUCTR2007-001838-13-GB   Australia;Brazil;United Kingdom;United States
Reminder product
   Amicus Therapeutics, Inc.
      2010   Phase 3   EUCTR2009-013459-31-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      2010   Phase 3   EUCTR2009-013459-31-BE   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
      -   -   EUCTR2009-013459-31-DE   Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom
Replagal
   AMICUS THERAPEUTICS, INC
      2011   -   EUCTR2010-022636-37-IT   Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2012   -   EUCTR2010-022709-16-GB   Belgium;United Kingdom
      2012   -   EUCTR2010-022636-37-DE   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   Phase 2   EUCTR2010-022709-16-BE   Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom
      2011   Phase 3   EUCTR2010-022636-37-GR   Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-GB   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-DK   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-BE   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
      2011   -   EUCTR2010-022636-37-AT   Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States
   Baylor Research Institute
      2003   Phase 2   NCT00068107   United States
   National Institute of Neurological Disorders and Stroke (NINDS)
      2004   Phase 4   NCT00097890   United States
      2004   Phase 2   NCT00075244   United States
      2003   Phase 2   NCT00071877   United States
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
   Sanofi Aventis Groupe (SAG)
      2019   Phase 4   EUCTR2019-000064-21-GB   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
      -   Phase 4   EUCTR2019-000064-21-NO   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
      -   Phase 4   EUCTR2019-000064-21-CZ   Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom
   Shire
      2022   Phase 4   NCT05067868   India
      2021   Phase 3   NCT04840667   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2011   Phase 2   NCT01363492   United States
      2010   Phase 3   NCT01124643   Australia;Czech Republic;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
      2008   Phase 3   NCT00864851   Australia;Czech Republic;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
   Shire Human Genetic Therapies (HGT), Inc.
      2012   Phase 3;Phase 4   EUCTR2009-015985-75-SI   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
      2011   Phase 3;Phase 4   EUCTR2009-015985-75-GB   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
      2011   -   EUCTR2009-015985-75-FI   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
      2010   -   EUCTR2009-015985-75-PL   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
      2010   -   EUCTR2009-015985-75-CZ   Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States
   Shire Human Genetic Therapies, Inc.
      2022   Phase 3   EUCTR2018-004689-32-FI   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-SE   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PT   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PL   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-GR   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
   Shire Human Genetic Therapies, Inc. (Shire HGT)
      2011   -   EUCTR2007-005543-22-SI   Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States
      2010   Phase 3;Phase 4   EUCTR2007-005543-22-GB   Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States
      2010   Phase 3;Phase 4   EUCTR2007-005543-22-FI   Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States
      2008   -   EUCTR2007-005543-22-CZ   Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States
   TKT Inc
      2004   -   EUCTR2004-000772-14-CZ   Czech Republic
   Takeda
      2022   Phase 3   NCT04974749   China
   Takeda Development Center Americas, Inc
      -   Phase 3   EUCTR2022-004246-35-Outside-EU/EEA   China
   University Hospital, Rouen
      2017   -   NCT02956954   France
   sanofi-aventis recherche et développement
      2022   Phase 3   EUCTR2021-002320-20-PL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NO   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-DK   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-CZ   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-AT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002320-20-GR   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-002320-20-ES   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
Replagal agalsidase alfa
   National Institute of Neurological Disorders and Stroke (NINDS)
      2003   Phase 1   NCT00357786   United States
RGX-111
   REGENXBIO Inc.
      2019   Phase 1/Phase 2   NCT03580083   Brazil;Israel;United States
RGX-121
   REGENXBIO Inc.
      2021   Phase 1/Phase 2   NCT04571970   Canada;United States
      2018   Phase 2/Phase 3   NCT03566043   Brazil;United States
RH growth hormone
   National Human Genome Research Institute (NHGRI)
      2014   Phase 1/Phase 2   NCT02124070   United States
Rharsa
   Assistance Publique - Hôpitaux de Paris
      2008   Phase 2   NCT01303146   France
Rhasa
   SHIRE HUMAN GENETIC THERAPIES, INC
      2020   Phase 2   EUCTR2018-003291-12-IT   Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States
   Shire
      2007   Phase 1/Phase 2   NCT00633139   Denmark
   Shire Human Genetic Therapies Inc
      2010   Phase 2   EUCTR2008-000084-41-BE   Belgium;Denmark;France;Italy
      2009   Phase 2   EUCTR2008-000084-41-IT   Belgium;Denmark;France;Italy
      2009   Phase 2   EUCTR2008-000084-41-FR   Belgium;Denmark;France;Italy
      2008   Phase 2   EUCTR2008-000084-41-DK   Belgium;Denmark;France;Italy
   Shire Human Genetics Therapies Inc
      2017   Phase 1;Phase 2   EUCTR2012-003775-20-IT   Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom
      2016   Phase 1;Phase 2   EUCTR2012-003775-20-GB   Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom
      2014   Phase 1;Phase 2   EUCTR2012-003775-20-CZ   Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom
      2012   Phase 1;Phase 2   EUCTR2012-003775-20-FR   Argentina;Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;United Kingdom
      2012   Phase 1;Phase 2   EUCTR2011-002044-28-DE   Australia;Brazil;Denmark;France;Germany;Japan
      2011   Phase 1;Phase 2   EUCTR2011-002044-28-DK   Australia;Brazil;Denmark;France;Germany;Japan
   Shire Pharmaceutical Ireland Limited
      2008   -   EUCTR2007-006345-40-DK   Denmark
   Shire Pharmaceuticals Ireland Limited
      2008   Phase 2   EUCTR2007-007165-20-DK   Denmark
      2006   -   EUCTR2006-005341-11-DK   Denmark
Rhasa - dose level 1
   Shire
      2007   Phase 1   NCT00418561   Denmark
Rhasa - dose level 2
   Shire
      2007   Phase 1   NCT00418561   Denmark
Rhasa - dose level 3
   Shire
      2007   Phase 1   NCT00418561   Denmark
Rhasa [recombinant human arylsulfatase A], formerly HGT-1110, NOW also known AS TAK-611
   Shire Human Genetics Therapies Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited
      2013   Phase 1;Phase 2   EUCTR2012-003775-20-DK   Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2012-003775-20-DE   Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom
Rhasm
   Genzyme Corporation
      2019   Phase 2   EUCTR2013-000051-40-BE   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2018   Phase 2   EUCTR2013-000051-40-DE   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-PT   Argentina;Australia;Belgium;Brazil;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2016   Phase 2;Phase 3   EUCTR2015-000371-26-NL   Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2016   Phase 2   EUCTR2013-000051-40-IT   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2;Phase 3   EUCTR2015-000371-26-GB   Argentina;Australia;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   -   EUCTR2015-000371-26-ES   Argentina;Australia;Brazil;Chile;France;Germany;Israel;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   Phase 2;Phase 3   EUCTR2015-000371-26-DE   Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-IT   Brazil;Chile;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-GB   Brazil;France;Germany;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003198-40-DE   Brazil;France;Germany;Italy;United Kingdom;United States
      2014   -   EUCTR2010-023953-12-IT   Brazil;Chile;France;Germany;Italy;Tunisia;United Kingdom;United States
      2013   Phase 2   EUCTR2013-000051-40-GB   Belgium;Brazil;France;Germany;Italy;United Kingdom;United States
      2013   -   EUCTR2010-023953-12-GB   Brazil;Chile;France;Germany;Italy;Tunisia;United Kingdom;United States
      2013   -   EUCTR2010-023953-12-FR   Brazil;Chile;France;Germany;Italy;Tunisia;United Kingdom;United States
      -   Phase 2   EUCTR2014-003198-40-FR   Brazil;Chile;France;Germany;Italy;United Kingdom;United States
   Genzyme, a Sanofi Company
      2006   Phase 1   NCT00410566   United States
Rhgalns
   BIOMARIN PHARMACEUTICAL INC.
      2012   Phase 2   EUCTR2011-003197-84-IT   Italy;United Kingdom;United States
      2011   -   EUCTR2010-020199-45-IT   Argentina;Australia;Brazil;Canada;Colombia;Denmark;Germany;Italy;Japan;Korea, Democratic People's Republic of;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   -   EUCTR2010-020198-18-IT   Denmark;Germany;Italy;Norway;Portugal;United Kingdom
   BioMarin Pharmaceutical Inc
      2012   Phase 3   EUCTR2010-020198-18-NO   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States
      2011   -   EUCTR2010-020198-18-PT   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States
      2011   -   EUCTR2010-020198-18-DK   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States
      2011   -   EUCTR2010-020198-18-DE   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States
      2010   -   EUCTR2010-020198-18-GB   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2010-020198-18-NL   Denmark;France;Germany;Italy;Netherlands;Norway;Portugal;United Kingdom
      -   Phase 3   EUCTR2010-020198-18-FR   Denmark;France;Germany;Italy;Netherlands;Norway;Portugal;United Kingdom
   BioMarin Pharmaceutical Inc.
      2013   Phase 3   EUCTR2010-020199-45-ES   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States
      2012   -   EUCTR2011-005703-33-GB   Germany;Italy;United Kingdom;United States
      2012   -   EUCTR2011-005703-33-DE   Germany;Italy;United Kingdom;United States
      2012   -   EUCTR2011-005682-20-GB   Canada;Germany;United Kingdom;United States
      2012   -   EUCTR2011-005682-20-DE   Canada;Germany;United Kingdom;United States
      2012   -   EUCTR2010-020199-45-PT   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2012   Phase 3   EUCTR2010-020199-45-DK   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States
      2012   Phase 3   EUCTR2010-020199-45-DE   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2011   Phase 2   EUCTR2011-003197-84-GB   Italy;United Kingdom;United States
      2011   -   EUCTR2010-020199-45-GB   Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;United Kingdom;United States
      2010   -   EUCTR2010-021048-16-GB   United Kingdom
      2009   -   EUCTR2008-007365-23-GB   United Kingdom
Rhgus
   Hamazaki Takashi
      2020   Phase 3   JPRN-jRCT2051190085   -
   Ultragenyx Pharmaceutical Inc.
      2016   Phase 3   EUCTR2015-001875-32-PT   Brazil;Mexico;Portugal;United States
      2016   Phase 2   EUCTR2015-000104-26-PT   Portugal;Spain;United States
      2016   Phase 2   EUCTR2015-000104-26-ES   Portugal;Spain;United States
      2014   Phase 1;Phase 2   EUCTR2013-001152-35-ES   Spain;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2013-001152-35-GB   Spain;United Kingdom
      -   -   EUCTR2015-001875-32-Outside-EU/EEA   United States
      -   -   EUCTR2015-000104-26-Outside-EU/EEA   United States
      -   -   EUCTR2014-005638-71-Outside-EU/EEA   United States
Rhhns
   SHIRE HUMAN GENETIC THERAPIES, INC
      2015   Phase 2   EUCTR2014-003960-20-IT   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire
      2014   Phase 2   NCT02060526   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2010   Phase 1/Phase 2   NCT01155778   Netherlands;United Kingdom
   Shire Human Genetic Therapies, Inc
      2014   Phase 2   EUCTR2013-003450-24-NL   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003450-24-IT   France;Germany;Italy;Netherlands;Spain;United Kingdom
      2014   Phase 2   EUCTR2013-003450-24-GB   Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003450-24-ES   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003450-24-DE   Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2011   Phase 1;Phase 2   EUCTR2010-021348-16-NL   Netherlands;United Kingdom
      2011   Phase 1;Phase 2   EUCTR2010-021348-16-GB   Netherlands;United Kingdom
      -   Phase 2   EUCTR2013-003450-24-FR   Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetic Therapies, Inc.
      2015   Phase 2   EUCTR2014-003960-20-NL   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003960-20-GB   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2015   Phase 2   EUCTR2014-003960-20-DE   France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetic Therapies, Inc. (Shire HGT)
      2010   Phase 1;Phase 2   EUCTR2009-015984-15-GB   United Kingdom
   Shire human Genetic Therapies, Inc.
      2015   Phase 2   EUCTR2014-003960-20-ES   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      -   Phase 2   EUCTR2014-003960-20-FR   Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
Rhhns-10 MG
   Shire
      2011   Phase 1/Phase 2   NCT01299727   Netherlands;United Kingdom
Rhhns-45 MG
   Shire
      2011   Phase 1/Phase 2   NCT01299727   Netherlands;United Kingdom
Rhhns-90 MG
   Shire
      2011   Phase 1/Phase 2   NCT01299727   Netherlands;United Kingdom
Rhlaman
   CHIESI FARMACEUTICI S.P.A.
      2018   Phase 2   EUCTR2016-001988-36-IT   Austria;Denmark;France;Germany;Italy
   Chiesi Farmaceutici S.p.A.
      2015   Phase 3   EUCTR2013-000336-97-DK   Denmark;France
      2013   Phase 3   EUCTR2013-000321-31-DK   Denmark;Poland
      -   Phase 3   EUCTR2013-000321-31-PL   Denmark;Poland
   Zymenex A/S
      2015   -   EUCTR2014-003950-15-DK   Denmark
      2013   Phase 3   EUCTR2013-000336-97-FR   Denmark;France
      2013   -   EUCTR2012-000979-17-SE   Belgium;Denmark;Germany;Spain;Sweden
      2013   Phase 3   EUCTR2012-000979-17-ES   Belgium;Denmark;Germany;Spain;Sweden
      2013   -   EUCTR2012-000979-17-BE   Belgium;Denmark;Germany;Spain;Sweden
      2012   -   EUCTR2012-000979-17-DK   Belgium;Denmark;Germany;Spain;Sweden
      2012   -   EUCTR2012-000979-17-DE   Belgium;Denmark;Germany;Spain;Sweden
      2012   -   EUCTR2011-004355-40-GB   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   -   EUCTR2011-004355-40-ES   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   Phase 2   EUCTR2011-004355-40-DK   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   -   EUCTR2011-004355-40-DE   Belgium;Denmark;Germany;Spain;United Kingdom
      2012   -   EUCTR2011-004355-40-BE   Belgium;Denmark;Germany;Spain;United Kingdom
      2010   -   EUCTR2010-022085-26-DK   Denmark
      2010   -   EUCTR2010-022084-36-DK   Denmark
Rhnaglu
   Alexion Pharmaceuticals, Inc
      2015   Phase 2   EUCTR2015-001983-20-GB   United Kingdom
   Alexion Pharmaceuticals, Inc.
      2016   Phase 1   EUCTR2013-003400-39-ES   Spain;United Kingdom;United States
      2014   Phase 1;Phase 2   EUCTR2013-003400-39-GB   Spain;United Kingdom;United States
Rhnaglu radiolabelled
   Alexion Pharmaceuticals, Inc.
      2016   Phase 2   EUCTR2015-000753-20-NL   Netherlands
Rhnaglu-IGF2
   Allievex Corporation
      2022   Phase 3;Phase 4   EUCTR2022-002762-33-DE   Colombia;Germany;Turkey;United Kingdom;United States
      2018   Phase 2   EUCTR2017-003083-13-GB   Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-001985-25-DE   Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom
      2015   Phase 1;Phase 2   EUCTR2015-001985-25-GB   Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom
      -   Phase 2   EUCTR2017-003083-13-DE   Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States
   BioMarin Pharmaceutical Inc.
      2017   Phase 2   EUCTR2017-003083-13-ES   Argentina;Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-001985-25-ES   Australia;Brazil;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom
Rimiducid
   Bellicum Pharmaceuticals
      2017   -   NCT03639844   United States
Rituximab
   Alice Bertaina
      2023   Phase 1/Phase 2   NCT05508009   United States
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
   OSPEDALE SAN RAFFAELE
      2018   Phase 1   EUCTR2017-002430-23-IT   Italy
   Takeda
      2023   Phase 4   NCT05494593   United States
Rivogenlecleucel
   Bellicum Pharmaceuticals
      2017   -   NCT03639844   United States
RP 103
   HOSPICES CIVILS DE LYON
      2010   -   EUCTR2010-020098-18-FR   France
RP103
   Amgen
      2013   Phase 3   NCT01733316   Belgium;France;Italy;Netherlands;United Kingdom;United States
      2012   Phase 3   NCT01744782   Brazil;United States
   Horizon Pharma USA, Inc.
      2014   Phase 3   EUCTR2012-002773-64-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2013   Phase 3   EUCTR2012-002773-64-NL   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2010   Phase 3   NCT01000961   France;Netherlands;United States
      2010   Phase 3   EUCTR2010-018365-34-NL   France;Netherlands;United States
      2009   Phase 1/Phase 2   NCT00872729   United States
   Raptor Therapeutics Inc.
      2014   Phase 3   EUCTR2012-002773-64-IT   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 3   EUCTR2012-002773-64-BE   Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States
      2013   Phase 3   EUCTR2012-002773-64-FR   Belgium;France;Italy;Netherlands;United Kingdom;United States
      2010   -   EUCTR2009-017882-42-NL   France;Netherlands
      2010   Phase 3   EUCTR2009-017882-42-FR   France;Netherlands
      -   Phase 3   EUCTR2010-018365-34-FR   France;Netherlands
RVX000222
   Resverlogix Corp
      2022   Phase 1/Phase 2   NCT03228940   Canada
SAF-301
   LYSOGENE
      2013   Phase 1/Phase 2   NCT02053064   France
      2011   Phase 1/Phase 2   NCT01474343   France
   SANFILIPPO Therapeutics SAS
      -   -   EUCTR2010-019962-10-FR   France
Saline solution FOR injection
   Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
      2017   Phase 1/Phase 2   NCT03153319   United States
SAR402671, GZ402671 or GZ / SAR402671
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2023   Phase 3   EUCTR2021-005402-10-IT   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
   Sanofi-Aventis Recherche & Développement
      2022   Phase 3   EUCTR2021-002350-90-PL   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-NO   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-FI   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-AT   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DK   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DE   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
   Sanofi-aventis recherche & développement
      2022   Phase 3   EUCTR2021-005402-10-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005402-10-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Türkiye;United Kingdom;United States
SAR402671, GZ402671 or GZ/SAR402671
   Genzyme Corporation
      2015   Phase 2   EUCTR2014-004995-49-PL   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
      2015   Phase 2   EUCTR2014-004995-49-GB   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
      2015   Phase 2   EUCTR2014-004995-49-FR   Czech Republic;France;Poland;Russian Federation;United Kingdom;United States
   Sanofi-Aventis Recherche & Développement
      2022   Phase 3   EUCTR2021-002350-90-RO   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-PL   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-NO   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-FR   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-FI   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-AT   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2021   Phase 3   EUCTR2021-002350-90-GR   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DK   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DE   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
   sanofi-aventis recherche et développement
      2022   Phase 3   EUCTR2021-002320-20-PL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NO   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-DK   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-CZ   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-AT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002320-20-GR   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-002320-20-ES   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
SB-318
   Sangamo Therapeutics
      2020   -   NCT04628871   United States
      2017   Phase 1/Phase 2   NCT02702115   United States
SB-47171
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000206-28-GB   United Kingdom;United States
      2018   Phase 1;Phase 2   EUCTR2018-000192-33-GB   United Kingdom;United States
SB-47898
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000206-28-GB   United Kingdom;United States
      2018   Phase 1;Phase 2   EUCTR2018-000192-33-GB   United Kingdom;United States
SB-913
   Sangamo Therapeutics
      2020   -   NCT04628871   United States
      2017   Phase 1/Phase 2   NCT03041324   United States
SB-A6P-HRL donor vector
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000206-28-GB   United Kingdom;United States
SB-FIX
   Sangamo Therapeutics
      2020   -   NCT04628871   United States
SB-idua
   Sangamo Therapeutics, Inc.
      2018   Phase 1;Phase 2   EUCTR2018-000206-28-GB   United Kingdom;United States
SBC-102
   Alexion Pharmaceuitcals Inc
      2014   Phase 2   EUCTR2011-004287-30-HR   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-DE   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals
      2011   Phase 2/Phase 3   NCT01473875   France;United Kingdom
      2011   Phase 2/Phase 3   NCT01371825   Egypt;France;Germany;India;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals Inc
      2015   Phase 2   EUCTR2014-000533-22-FI   Finland;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2011-004287-30-NL   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2015   Phase 2   EUCTR2011-004287-30-GB   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2014-000533-22-GB   Finland;Italy;United Kingdom;United States
   Alexion Pharmaceuticals inc
      2014   Phase 2   EUCTR2011-004287-30-DK   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals, Inc.
      2015   Phase 3   EUCTR2011-002750-31-CZ   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-GB   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-DE   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      -   Phase 3   EUCTR2011-002750-31-PL   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
   SYNAGEVA BIOPHARMA CORP.
      2012   -   EUCTR2011-000032-28-IT   Germany;Ireland;Italy;Taiwan;United Kingdom;United States
   Synageva BioPharma Corp.
      2014   Phase 2   EUCTR2014-000533-22-IT   Finland;Italy;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-IT   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-ES   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-BE   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
   Synageva BioPharma Corporation
      2013   Phase 3   EUCTR2011-002750-31-IT   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-HR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-GR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-ES   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      -   Phase 3   EUCTR2011-002750-31-FR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
   Synageva Biopharma Corp.
      2011   -   EUCTR2011-002880-42-GB   France;Germany;United Kingdom;United States
      2011   Phase 2   EUCTR2011-001513-13-GB   Czech Republic;France;United Kingdom;United States
      2011   Phase 2   EUCTR2011-001513-13-CZ   Czech Republic;France;United Kingdom;United States
      2011   Phase 1;Phase 2;Phase 3   EUCTR2011-000032-28-GB   France;Germany;Ireland;Italy;Taiwan;United Kingdom;United States
      2011   -   EUCTR2010-024068-16-GB   Czech Republic;United Kingdom
      2011   -   EUCTR2010-024068-16-CZ   Czech Republic;United Kingdom;United States
      -   Phase 2   EUCTR2011-002880-42-FR   France;Germany;United Kingdom;United States
      -   Phase 1;Phase 2   EUCTR2011-000032-28-FR   France;Germany;Ireland;Italy;United Kingdom;United States
SBC-102, recombinant human lysosomal acid lipase
   Alexion Pharmaceuticals Inc.
      2013   Phase 2;Phase 3   EUCTR2011-000032-28-IE   Egypt;France;Germany;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States
   Synageva Biopharma Corp.
      2013   -   EUCTR2011-000032-28-DE   France;Germany;Italy;Taiwan;United Kingdom;United States
SBC-103
   Alexion Pharmaceuticals
      2015   Phase 1/Phase 2   NCT02618512   United Kingdom
      2015   Phase 1/Phase 2   NCT02324049   Spain;United Kingdom;United States
   Alexion Pharmaceuticals, Inc
      2015   Phase 2   EUCTR2015-001983-20-GB   United Kingdom
   Alexion Pharmaceuticals, Inc.
      2016   Phase 1   EUCTR2013-003400-39-ES   Spain;United Kingdom;United States
      2014   Phase 1;Phase 2   EUCTR2013-003400-39-GB   Spain;United Kingdom;United States
Scaav9.CB.CLN6
   Amicus Therapeutics, Inc.
      2021   Phase 1;Phase 2   EUCTR2021-000706-21-DE   Germany;United States
Scaav9.U1A.hsgsh
   Abeona Therapeutics Inc
      2020   Phase 1;Phase 2   EUCTR2019-002979-34-ES   Australia;France;Germany;Spain;United States
      2019   Phase 1;Phase 2   EUCTR2015-003904-21-FR   Australia;Brazil;France;Germany;Spain;United Kingdom;United States
Scaav9.U1A.sgsh
   Abeona Therapeutics Inc
      2019   Phase 1;Phase 2   EUCTR2018-000504-42-ES   Australia;Spain;United States
      2016   Phase 1;Phase 2   EUCTR2015-003904-21-ES   Australia;Spain;United States
Sebelipase alfa
   Alexion Pharmaceuitcals Inc
      2014   Phase 2   EUCTR2011-004287-30-HR   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-DE   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals
      2015   -   NCT02376751   -
      2014   Phase 2   NCT02193867   Finland;Italy;United Kingdom;United States
      2014   Phase 2   NCT02112994   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2013   Phase 3   NCT01757184   Argentina;Australia;Brazil;Canada;Croatia;Czech Republic;Czechia;France;Germany;Greece;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2011   Phase 2   NCT01488097   Canada;Czech Republic;Czechia;France;United Kingdom;United States
      2011   Phase 2/Phase 3   NCT01371825   Egypt;France;Germany;India;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals Inc
      2015   Phase 2   EUCTR2014-000533-22-FI   Finland;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2011-004287-30-NL   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2015   Phase 2   EUCTR2011-004287-30-GB   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2014-000533-22-GB   Finland;Italy;United Kingdom;United States
   Alexion Pharmaceuticals Inc.
      2013   Phase 2;Phase 3   EUCTR2011-000032-28-IE   Egypt;France;Germany;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals inc
      2014   Phase 2   EUCTR2011-004287-30-DK   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals, Inc.
      2015   Phase 3   EUCTR2011-002750-31-CZ   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-GB   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-DE   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      -   Phase 3   EUCTR2011-002750-31-PL   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
   Synageva BioPharma Corp.
      2014   Phase 2   EUCTR2014-000533-22-IT   Finland;Italy;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-IT   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-BE   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
   Synageva BioPharma Corporation
      2013   Phase 3   EUCTR2011-002750-31-IT   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-HR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-GR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-ES   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      -   Phase 3   EUCTR2011-002750-31-FR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
   Synageva Biopharma Corp.
      2013   -   EUCTR2011-000032-28-DE   France;Germany;Italy;Taiwan;United Kingdom;United States
      2011   Phase 2   EUCTR2011-001513-13-CZ   Czech Republic;France;United Kingdom;United States
Sebelipase alfa 0.35 MG/KG
   Alexion Pharmaceuticals
      2011   Phase 1/Phase 2   NCT01307098   Czech Republic;Czechia;France;United Kingdom;United States
Sebelipase alfa 1 MG/KG
   Alexion Pharmaceuticals
      2011   Phase 1/Phase 2   NCT01307098   Czech Republic;Czechia;France;United Kingdom;United States
Sebelipase alfa 3 MG/KG
   Alexion Pharmaceuticals
      2011   Phase 1/Phase 2   NCT01307098   Czech Republic;Czechia;France;United Kingdom;United States
SHP611
   SHIRE HUMAN GENETIC THERAPIES, INC
      2020   Phase 2   EUCTR2018-003291-12-IT   Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States
   Shire
      2019   Phase 2   NCT03771898   Argentina;Belgium;Brazil;Canada;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetic Therapies, Inc.
      2021   Phase 2   EUCTR2018-003291-12-GR   Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States
      2020   Phase 2   EUCTR2018-003291-12-DE   Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-NL   Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-GB   Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-FR   Belgium;Brazil;Canada;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-ES   Belgium;Brazil;Canada;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003291-12-BE   Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Spain;United Kingdom;United States
   Shire Human Genetics Therapies Inc
      2017   Phase 1;Phase 2   EUCTR2012-003775-20-IT   Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom
      2016   Phase 1;Phase 2   EUCTR2012-003775-20-GB   Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom
      2014   Phase 1;Phase 2   EUCTR2012-003775-20-CZ   Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom
   Shire Human Genetics Therapies Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited
      2013   Phase 1;Phase 2   EUCTR2012-003775-20-DK   Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2012-003775-20-DE   Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom
SHP675
   Shire Human Genetic Therapies, Inc.
      2022   Phase 3   EUCTR2018-004689-32-FI   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-SE   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PT   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PL   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-GR   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
Sirolimus
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
   Prevail Therapeutics
      2021   Phase 1/Phase 2   NCT04411654   United Kingdom;United States
Skeletal survey
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Skin biopsy
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
Slit lamp assessment and intra-ocular pressure measurement
   Université de Montréal
      2014   -   NCT02023086   Canada
SOBI003
   Swedish Orphan Biovitrum
      2019   Phase 1/Phase 2   NCT03811028   Turkey;United States
      2018   Phase 1/Phase 2   NCT03423186   Germany;Turkey;United States
   Swedish Orphan Biovitrum AB (publ)
      2019   Phase 1   EUCTR2017-002806-10-NL   Germany;Netherlands;Turkey;United States
      -   Phase 1   EUCTR2017-002806-10-DE   Germany;Netherlands;Turkey;United States
Somatropin
   Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
      2008   Phase 2/Phase 3   NCT00748969   United States
Speech and modified barium swallow study
   National Human Genome Research Institute (NHGRI)
      2019   Phase 1/Phase 2   NCT03952637   United States
ST-920
   Sangamo Therapeutics
      2021   -   NCT05039866   Australia;Canada;United Kingdom;United States
      2019   Phase 1/Phase 2   NCT04046224   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
   Sangamo Therapeutics, Inc.
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-IT   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
      2022   Phase 1;Phase 2   EUCTR2019-000667-24-DE   Australia;Canada;Germany;Italy;Taiwan;United Kingdom;United States
      2019   Phase 1;Phase 2   EUCTR2019-000667-24-GB   United Kingdom;United States
Stem cell transplant
   Masonic Cancer Center, University of Minnesota
      2004   Phase 2   NCT00176891   United States
      1999   Phase 2   NCT00176917   United States
      1995   Phase 2/Phase 3   NCT00176904   United States
Stem cell transplantation
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States
      2008   Phase 2   NCT00668564   United States
SUB195712
   IntraBio Ltd
      2019   Phase 2   EUCTR2018-004406-25-GB   Germany;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004331-71-GB   Germany;Slovakia;Spain;United Kingdom;United States
TAK-675
   Shire Human Genetic Therapies, Inc.
      2022   Phase 3   EUCTR2018-004689-32-FI   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-SE   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PT   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-PL   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
      2021   Phase 3   EUCTR2018-004689-32-GR   Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine
   Takeda Development Center Americas, Inc
      -   Phase 3   EUCTR2022-004246-35-Outside-EU/EEA   China
Taliglucerase alfa
   Pfizer
      2011   Phase 3   NCT01422187   -
      2011   Phase 3   NCT01411228   Israel;Paraguay;South Africa
      2010   Phase 4   NCT01132690   Israel;Paraguay;South Africa;United States
      2008   Phase 3   NCT00712348   Australia;Canada;Israel;Serbia;Spain;United Kingdom;United States
      2008   Phase 3   NCT00705939   Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States
   Protalix Biotherapeutics
      2012   Phase 3   EUCTR2011-003685-32-ES   Canada;Chile;Israel;Italy;Mexico;Serbia;South Africa;Spain
      2011   Phase 3   EUCTR2011-003676-37-ES   Israel;South Africa;Spain
      2009   Phase 3   EUCTR2008-005826-35-GB   Italy;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005825-12-GB   Germany;Italy;Spain;United Kingdom
      2009   -   EUCTR2008-005825-12-DE   Germany;Italy;Spain;United Kingdom
Taliglucerase alfa - recombinant human glucocerebrosidase
   Protalix Biotherapeutics
      -   -   EUCTR2008-005826-35-Outside-EU/EEA   Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States
      -   -   EUCTR2008-005825-12-Outside-EU/EEA   Canada;Israel;United Kingdom;United States
      -   -   EUCTR2007-000498-42-Outside-EU/EEA   Israel;United States
TBD
   BioMarin Pharmaceutical Inc.
      2015   -   EUCTR2014-003480-37-IT   Germany;Italy;Japan;United Kingdom;United States
      2014   Phase 1;Phase 2   EUCTR2012-005430-11-IT   Germany;Italy;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2012-005430-11-DE   Germany;United Kingdom
Temperature sensitivity measurement with advanced thermal stimulation
   Universitaire Ziekenhuizen Leuven
      2014   Phase 0   NCT02533076   Belgium
Thiotepa--escalated dose
   University of Florida
      2018   Phase 1/Phase 2   NCT03513328   United States
Thiotepa--single daily dose
   University of Florida
      2018   Phase 1/Phase 2   NCT03513328   United States
Tividenofusp alfa
   Denali Therapeutics Inc.
      2023   Phase 2/Phase 3   NCT06075537   Belgium;Canada;Czechia;France;Germany;Netherlands;Spain;United States
      2022   Phase 2/Phase 3   NCT05371613   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czechia;France;Germany;Italy;Netherlands;Spain;Sweden;Turkey;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-SE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-NL   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-005200-35-BE   Argentina;Australia;Belgium;Brazil;Canada;Colombia;Czech Republic;Czechia;France;Germany;Italy;Mexico;Netherlands;Spain;Sweden;Türkiye;United Kingdom;United States
      2020   Phase 1/Phase 2   NCT04251026   Canada;Netherlands;United Kingdom;United States
Total body irradiation
   Alice Bertaina
      2023   Phase 1/Phase 2   NCT05508009   United States
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States
Total body irradiation with marrow boosting
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
Tralesinidase alfa
   Allievex Corporation
      2022   Phase 3;Phase 4   EUCTR2022-002762-33-DE   Colombia;Germany;Turkey;United Kingdom;United States
Transduced CD34+ hematopoietic stem cell
   Shenzhen Second People's Hospital
      2015   Phase 1/Phase 2   NCT02559830   China
Trappsol cyclo
   CTD Holdings, Inc.
      2016   Phase 1;Phase 2   EUCTR2015-005761-23-GB   Italy;Sweden;United Kingdom
   Cyclo Therapeutics, Inc.
      2017   Phase 1;Phase 2   EUCTR2015-005761-23-SE   Italy;Sweden;United Kingdom
Treosulfan
   Tokai University School of Medicine
      2013   Phase 1   JPRN-UMIN000009758   Japan
Triapin& triapin mite
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Tropicamide
   Université de Montréal
      2014   -   NCT02023086   Canada
TSHA-101
   Dr. Anupam Sehgal
      2021   Phase 1/Phase 2   NCT04798235   Canada
TTX-381
   Tern Therapeutics, LLC
      2023   Phase 1/Phase 2   NCT05791864   United Kingdom
Unrelated umbilical cord blood transplant
   Duke University
      2008   Phase 1   NCT00744692   United States
Uplyso
   Protalix Biotherapeutics
      2009   Phase 3   EUCTR2008-005826-35-GB   Italy;Spain;United Kingdom
      2009   Phase 3   EUCTR2008-005825-12-GB   Germany;Italy;Spain;United Kingdom
      2009   -   EUCTR2008-005825-12-DE   Germany;Italy;Spain;United Kingdom
Urine collection
   Baylor Research Institute
      2018   -   NCT04002531   United States
Usan: sebelipasa alfa
   Synageva BioPharma Corp.
      2014   Phase 2   EUCTR2011-004287-30-ES   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
Usan: sebelipase alpha
   Alexion Pharmaceuitcals Inc
      2014   Phase 2   EUCTR2011-004287-30-HR   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-DE   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals Inc
      2015   Phase 2   EUCTR2014-000533-22-FI   Finland;Italy;United Kingdom;United States
      2015   Phase 2   EUCTR2011-004287-30-NL   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
      2015   Phase 2   EUCTR2011-004287-30-GB   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2014-000533-22-GB   Finland;Italy;United Kingdom;United States
   Alexion Pharmaceuticals inc
      2014   Phase 2   EUCTR2011-004287-30-DK   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
   Alexion Pharmaceuticals, Inc.
      2015   Phase 3   EUCTR2011-002750-31-CZ   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-GB   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      2013   Phase 3   EUCTR2011-002750-31-DE   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
      -   Phase 3   EUCTR2011-002750-31-PL   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States
   Synageva BioPharma Corp.
      2014   Phase 2   EUCTR2014-000533-22-IT   Finland;Italy;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-IT   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2011-004287-30-BE   Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States
   Synageva BioPharma Corporation
      2013   Phase 3   EUCTR2011-002750-31-IT   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-HR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-GR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      2013   Phase 3   EUCTR2011-002750-31-ES   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
      -   Phase 3   EUCTR2011-002750-31-FR   Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom
UX003
   Ultragenyx Pharmaceutical Inc
      2015   Phase 3   NCT02432144   Brazil;Mexico;Portugal;United States
      2015   Phase 2   NCT02418455   Portugal;Spain;United States
      2014   Phase 3   NCT02230566   United States
   Ultragenyx Pharmaceutical Inc.
      2016   Phase 3   EUCTR2015-001875-32-PT   Brazil;Mexico;Portugal;United States
      2016   Phase 2   EUCTR2015-000104-26-PT   Portugal;Spain;United States
      2016   Phase 2   EUCTR2015-000104-26-ES   Portugal;Spain;United States
      2014   Phase 1;Phase 2   EUCTR2013-001152-35-ES   Spain;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2013-001152-35-GB   Spain;United Kingdom
      -   -   EUCTR2015-001875-32-Outside-EU/EEA   United States
      -   -   EUCTR2015-000104-26-Outside-EU/EEA   United States
      -   -   EUCTR2014-005638-71-Outside-EU/EEA   United States
UX111
   Ultragenyx Pharmaceutical Inc
      2016   Phase 2/Phase 3   NCT02716246   Australia;France;Germany;Spain;United States
Vector viral adenoasociado DE serotipo 9 QUE contiene EL GEN DE LA sulfamidasa humana
   Laboratorios del Dr. Esteve, S.A.
      2016   Phase 1;Phase 2   EUCTR2015-000359-26-ES   Spain
Velaglucerase alfa
   Baylor Research Institute
      2015   Phase 4   NCT02528617   United States
   SHIRE HUMAN GENETIC THERAPIES, INC
      2018   Phase 4   EUCTR2015-001578-17-IT   Germany;Israel;Italy;Spain;United Kingdom;United States
   Shire
      2016   Phase 4   NCT02574286   Israel;Spain;United Kingdom;United States
      2013   Phase 3   NCT01842841   Japan
      2012   Phase 1/Phase 2   NCT01685216   Egypt;India;Tunisia
      2012   Phase 3   NCT01614574   Japan
      2009   -   NCT00954460   United States
      2008   Phase 3   NCT00553631   Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom;United States
      2007   Phase 2/Phase 3   NCT00478647   Israel;Poland;Spain;United Kingdom;United States
   Shire Human Genetic Therapies
      -   -   EUCTR2012-003427-38-Outside-EU/EEA   Egypt;India;Tunisia
   Shire Human Genetic Therapies Inc
      -   Phase 2;Phase 3   EUCTR2006-006304-11-DE   Germany;Italy;Spain;United Kingdom
   Shire Human Genetic Therapies, Inc.
      2008   -   EUCTR2008-001965-27-ES   Spain;United Kingdom
      2007   -   EUCTR2007-002840-21-GB   Italy;Spain;United Kingdom
   Takeda
      2023   Phase 3   NCT05529992   China
Velaglucerasi alfa
   SHIRE HUMAN GENETIC THERAPIES, INC
      2018   Phase 4   EUCTR2015-001578-17-IT   Germany;Israel;Italy;Spain;United Kingdom;United States
Velmanase alfa
   CHIESI FARMACEUTICI S.P.A.
      2018   Phase 2   EUCTR2016-001988-36-IT   Austria;Denmark;France;Germany;Italy
   Chiesi Farmaceutici S.p.A.
      2025   -   NCT06184503   United States
      2021   -   NCT04959240   -
      2021   Phase 3   NCT04031066   -
      2017   Phase 2   EUCTR2016-001988-36-FR   Austria;Denmark;France;Germany;Italy
      2017   Phase 2   EUCTR2016-001988-36-DE   Austria;Denmark;Germany
      2017   Phase 2   EUCTR2016-001988-36-AT   Austria;Denmark;Germany
      2016   Phase 2   NCT02998879   Austria;Denmark;France;Germany;Italy
      2016   Phase 2   EUCTR2016-001988-36-DK   Austria;Denmark;Germany;Italy
      2015   Phase 3   EUCTR2013-000336-97-DK   Denmark;France
      2013   Phase 3   EUCTR2013-000321-31-DK   Denmark;Poland
      -   Phase 3   EUCTR2013-000321-31-PL   Denmark;Poland
Venglustat
   GENZYME CORPORATION
      2020   Phase 3   EUCTR2019-002375-34-IT   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
   Genzyme Corporation
      2021   Phase 3   EUCTR2019-002375-34-AT   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-PT   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-GB   Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-DE   Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Türkiye;United Kingdom;United States
      2020   Phase 3   EUCTR2019-002375-34-CZ   Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
      2016   Phase 2   EUCTR2014-002550-39-DE   Germany;Japan;United Kingdom;United States
      -   Phase 3   EUCTR2019-002375-34-FR   Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
   Genzyme, a Sanofi Company
      2017   Phase 2   NCT02843035   Germany;Japan;United Kingdom;United States
   Sanofi
      2022   Phase 3   NCT05280548   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   NCT05222906   Argentina;Canada;China;France;Germany;Hungary;Italy;Japan;Turkey;United Kingdom;United States
      2022   Phase 3   NCT05206773   Argentina;Australia;Austria;Brazil;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Japan;Mexico;Norway;Poland;Romania;Switzerland;Turkey;United Kingdom;United States
   Sanofi-aventis recherche & développement
      -   Phase 3   EUCTR2021-005402-10-FR   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
   Tanaka Tomoyuki
      2023   Phase 3   JPRN-jRCT2021220029   Argentina;Canada;China;France;Germany;Hungary;Japan;United States
      2022   Phase 3   JPRN-jRCT2071220022   China;Italy;Japan;Republic of Korea
      2022   Phase 3   JPRN-jRCT2021220001   Argentina;Austria;Brazil;Canada;China;Denmark;Finland;Germany;Greece;Italy;Japan;Mexico;Norway;Poland;Romania;United Kingdom;United States
Venglustat GZ402671
   Genzyme, a Sanofi Company
      2020   Phase 3   NCT04221451   Argentina;Austria;Brazil;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
   Tanaka Tomoyuki
      2020   Phase 3   JPRN-jRCT2021200023   Argentina;Austria;Brazil;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States
Venglustat L-malate
   Sanofi-Aventis Recherche & Développement
      2022   Phase 3   EUCTR2021-002350-90-PL   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-NO   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-FI   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-AT   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2021   Phase 3   EUCTR2021-002350-90-GR   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DK   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DE   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
Venglustat malate
   Genzyme Corporation
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-FR   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      2021   Phase 2;Phase 3   EUCTR2020-003120-17-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
      -   Phase 2;Phase 3   EUCTR2020-003120-17-SE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States
   Sanofi-Aventis Recherche & Développement
      2022   Phase 3   EUCTR2021-002350-90-RO   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-PL   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-NO   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-FR   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-FI   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Turkey;Ukraine;United Kingdom
      2022   Phase 3   EUCTR2021-002350-90-AT   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      2021   Phase 3   EUCTR2021-002350-90-GR   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DK   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
      -   Phase 3   EUCTR2021-002350-90-DE   Argentina;Australia;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Switzerland;Türkiye;Ukraine;United Kingdom
   Sanofi-aventis recherche & développement
      2022   Phase 3   EUCTR2021-005402-10-HU   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005402-10-DE   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Türkiye;United Kingdom;United States
   sanofi-aventis recherche et développement
      2022   Phase 3   EUCTR2021-002320-20-PL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NO   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-NL   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-DK   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-CZ   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-AT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Türkiye;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002320-20-GR   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-002320-20-ES   Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
Venglustat malato
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2023   Phase 3   EUCTR2021-005402-10-IT   Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Italy;Japan;Taiwan;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002320-20-IT   Austria;Canada;China;Czech Republic;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States
      2021   Phase 3   EUCTR2021-002350-90-IT   Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom
Verenafusp alfa 12 MG/KG
   AO GENERIUM
      2023   Phase 1   NCT06475404   Russian Federation
Verenafusp alfa 6 MG/KG
   AO GENERIUM
      2023   Phase 1   NCT06475404   Russian Federation
Verenafusp alfa 9 MG/KG
   AO GENERIUM
      2023   Phase 1   NCT06475404   Russian Federation
Vestronidase alfa
   Hamazaki Takashi
      2020   Phase 3   JPRN-jRCT2051190085   -
VGN-R08B
   Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
      2023   Early Phase 1   NCT06272149   China
Visual field testing
   Université de Montréal
      2014   -   NCT02023086   Canada
Vital signs
   Baylor Research Institute
      2018   -   NCT04002531   United States
Vitamin D
   Shire
      2016   Phase 4   NCT02574286   Israel;Spain;United Kingdom;United States
Vitamin E
   Ain Shams University
      2023   Phase 3   NCT06211478   Egypt
Vosoritide
   University of California, San Francisco
      2023   Phase 1/Phase 2   NCT05845749   United States
Vpriv
   Shaare Zedek Medical Center
      2018   Phase 4   NCT03702361   Israel
      2016   Phase 4   NCT04120506   -
   Shire
      2008   Phase 3   NCT00635427   Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tunisia;United Kingdom;United States
      2007   Phase 3   NCT00430625   Argentina;Israel;Paraguay;Russian Federation;Tunisia;United States
   Shire Human Genetic Therapies
      -   -   EUCTR2012-003427-38-Outside-EU/EEA   Egypt;India;Tunisia
Vpriv - 400 U - polvere PER soluzione PER infusione - USO endovenoso - flaconcino(vetro) 1 flaconcino
   SHIRE HUMAN GENETIC THERAPIES, INC
      2018   Phase 4   EUCTR2015-001578-17-IT   Germany;Israel;Italy;Spain;United Kingdom;United States
Vpriv 400 units powder FOR solution OF infusion
   Shire Human Genetic Therapies, Inc.
      2016   Phase 4   EUCTR2015-001578-17-GB   Canada;Germany;Israel;Spain;United Kingdom;United States
      2016   Phase 4   EUCTR2015-001578-17-ES   Canada;Germany;Israel;Italy;Spain;United Kingdom;United States
      2016   Phase 4   EUCTR2015-001578-17-DE   Germany;India;Israel;Italy;Spain;Turkey;United Kingdom;United States
VTS-270
   Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company
      2020   Phase 2   NCT03687476   -
Warfarin
   The Cooper Health System
      2007   -   NCT00683189   United States
Water
   Koh KyuBum
      2023   Phase 3   JPRN-jRCT2051220125   Australia;France;Japan;Spain;USA
Whole body protein turnover
   University of Texas Southwestern Medical Center
      2006   -   NCT00286689   United States
Zavesca
   Actelion Pharmaceuticals Ltd
      2007   -   EUCTR2005-001651-37-HU   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2006   -   EUCTR2005-001651-37-ES   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2006   -   EUCTR2005-001651-37-DE   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2006   -   EUCTR2005-001651-37-CZ   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
      2005   Phase 4   EUCTR2005-001651-37-GB   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
   Actelion Pharmaceuticals Trading (Shanghai) Co., Ltd
      -   Phase 4   EUCTR2022-002514-16-Outside-EU/EEA   China
   Children's Research Institute
      2004   Phase 3   NCT00672022   United States
   Dipartimento di Pediatria Universita` di Napoli Federico II
      2006   -   EUCTR2006-005842-35-IT   Italy
   HOSPICES CIVILS DE LYON
      2006   -   EUCTR2006-004661-34-FR   France
   INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD
      2008   -   EUCTR2007-007164-20-ES   Spain
   ISTITUTO GIANNINA GASLINI
      2009   -   EUCTR2007-005516-61-IT   Italy
   Orphazyme ApS
      2015   Phase 1   EUCTR2014-005194-37-ES   Germany;Spain;Switzerland
      2015   Phase 1   EUCTR2014-005194-37-DE   Germany;Spain;Switzerland
Zavesca 84CPS 100MG
   Actelion Registration Ltd
      2006   -   EUCTR2005-001651-37-IT   Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom
Zestoretic 10,20
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Zestoric
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom
Zestril
   Salford Royal NHS Foundation Trust
      2009   Phase 4   EUCTR2007-007482-21-GB   United Kingdom