234. ペルオキシソーム病（副腎白質ジストロフィーを除く。） Disease details / Clinical trials / Drug dev / DR info

ABO-101
   Arbor Biotechnologies
      2025   Phase 1/Phase 2   NCT06839235   United States
Acei / angiotensin converting enzyme inhibitor
   Mayo Clinic
      2007   Phase 3   NCT00280215   United States
ALLN-177
   Allena Pharmaceuticals
      2018   Phase 2   NCT03391804   Germany;United Kingdom;United States
   Allena Pharmaceuticals, Inc.
      2018   Phase 2   EUCTR2017-003547-38-GB   Germany;United Kingdom;United States
      2018   Phase 2   EUCTR2017-003547-38-DE   Germany;United Kingdom;United States
ALN-65585
   Alnylam Pharmaceuticals, Inc.
      2018   Phase 2   EUCTR2016-003134-24-NL   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003134-24-GB   France;Germany;Israel;Netherlands;United Kingdom
      2017   Phase 2   EUCTR2016-003134-24-FR   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003134-24-DE   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States
      2017   Phase 1;Phase 2   EUCTR2015-004407-23-NL   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004407-23-GB   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States
      -   Phase 1;Phase 2   EUCTR2015-004407-23-DE   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States
ALN-GO1
   ALNYLAM PHARMACEUTICALS, INC.
      2021   Phase 3   EUCTR2019-001346-17-IT   Australia;Belgium;Egypt;France;Germany;India;Israel;Italy;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
   Alnylam Pharmaceuticals, Inc.
      2020   Phase 3   EUCTR2019-001346-17-GB   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
      2020   Phase 3   EUCTR2019-001346-17-FR   Australia;Belgium;Egypt;France;Germany;India;Israel;Italy;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
      2019   Phase 3   EUCTR2019-001346-17-NL   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
      2019   Phase 3   EUCTR2019-001346-17-BE   Australia;Belgium;Egypt;France;Germany;India;Israel;Italy;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
      2019   Phase 3   EUCTR2018-004014-17-GB   France;Germany;Israel;United Kingdom;United States
      2019   Phase 3   EUCTR2018-004014-17-FR   France;Germany;Israel;United Kingdom;United States
      2019   Phase 3   EUCTR2018-004014-17-DE   France;Germany;Israel;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001981-40-NL   France;Germany;Israel;Japan;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001981-40-GB   France;Germany;Israel;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001981-40-DE   France;Germany;Israel;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States
      2018   Phase 2   EUCTR2016-003134-24-NL   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003134-24-GB   France;Germany;Israel;Netherlands;United Kingdom
      2017   Phase 2   EUCTR2016-003134-24-FR   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003134-24-DE   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States
      2017   Phase 1;Phase 2   EUCTR2015-004407-23-NL   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004407-23-GB   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States
      -   Phase 1;Phase 2   EUCTR2015-004407-23-DE   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States
      -   Phase 3   EUCTR2019-001346-17-DE   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
ARB /angiotensin receptor blocker
   Mayo Clinic
      2007   Phase 3   NCT00280215   United States
B6-solution
   University of Cologne
      2010   -   EUCTR2009-015217-52-DE   Germany
Betaine
   Mayo Clinic
      2007   Phase 2   NCT00283387   United States
Blood samples collect
   Biocodex
      2024   Phase 3   NCT06465472   -
Campath-1H
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
Chenodeoxycholic acid
   Children's Hospital Research Foundation University of Cincinnati
      1997   -   NCT00004442   -
Cholic acid
   Children's Hospital Research Foundation University of Cincinnati
      1997   -   NCT00004442   -
   Department of Pediatric Gastroenterology, Academic Medical Centre
      -   Phase 2   EUCTR2010-022046-25-NL   Netherlands
Cholic acids
   Mirum Pharmaceuticals, Inc.
      1992   Phase 3   NCT00007020   United States
Clofarabine
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
Cyclosporine A
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
DCR-1171X
   Dicerna Pharmaceuticals, Inc.
      2016   Phase 1   EUCTR2015-003142-51-NL   France;Germany;Israel;Netherlands;United Kingdom;United States
      2016   Phase 1   EUCTR2015-003142-51-GB   France;Germany;Israel;Netherlands;United Kingdom;United States
DCR-L1360
   Dicerna Pharmaceuticals Inc
      2021   Phase 2   EUCTR2021-001083-16-PL   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2021-001083-16-ES   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2021-001083-16-DE   Canada;Germany;Italy;Japan;Lebanon;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2020-002826-97-FR   Australia;France;Germany;Italy;Japan;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-GB   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-FR   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-ES   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-DE   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Norway;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-PL   Australia;Canada;Egypt;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Saudi Arabia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-NL   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-GB   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-FR   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-ES   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-DE   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2018-003099-10-PL   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Norway;Poland;Romania;Spain;United Kingdom;United States
      -   Phase 1   EUCTR2017-003534-89-DE   France;Germany;Netherlands;United Kingdom;United States
   Dicerna Pharmaceuticals Inc . (a Novo Nordisk Company)
      2023   Phase 3   EUCTR2018-003099-10-NO   Australia;Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Netherlands;Norway;Poland;Romania;Spain;Türkiye;United Arab Emirates;United Kingdom;United States
   Dicerna Pharmaceuticals Inc.
      2021   Phase 2   EUCTR2021-001083-16-IT   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2020-002826-97-IT   Australia;France;Germany;Italy;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-IT   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Norway;Poland;Romania;Spain;United Kingdom;United States
   Dicerna Pharmaceuticals Inc. (a Novo Nordisk Company)
      2021   Phase 2   EUCTR2020-002826-97-DE   France;Germany;Italy;Lebanon;Morocco;Spain;United Arab Emirates;United Kingdom;United States
   Dicerna Pharmaceuticals, Inc.
      2020   Phase 1   EUCTR2020-000344-67-NL   France;Germany;Netherlands;United Kingdom;United States
      2020   Phase 1   EUCTR2020-000344-67-GB   France;Germany;Netherlands;United Kingdom;United States
      2020   Phase 1   EUCTR2020-000344-67-FR   France;Germany;Netherlands;United Kingdom;United States
      2020   Phase 1   EUCTR2020-000344-67-DE   France;Germany;Netherlands;United Kingdom;United States
DCR-PH1
   Dicerna Pharmaceuticals, Inc.
      2016   Phase 1   EUCTR2015-003142-51-NL   France;Germany;Israel;Netherlands;United Kingdom;United States
      2016   Phase 1   EUCTR2015-003142-51-GB   France;Germany;Israel;Netherlands;United Kingdom;United States
   Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
      2016   Phase 1   NCT02795325   Germany;Netherlands
DCR-phxc
   Dicerna Pharmaceuticals Inc
      2021   Phase 2   EUCTR2021-001083-16-PL   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2021-001083-16-ES   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2021-001083-16-DE   Canada;Germany;Italy;Japan;Lebanon;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2020-002826-97-FR   Australia;France;Germany;Italy;Japan;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-NL   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Norway;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-GB   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-FR   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-ES   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-DE   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Norway;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-PL   Australia;Canada;Egypt;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Saudi Arabia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-NL   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-GB   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-FR   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-ES   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-DE   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2018   Phase 1   EUCTR2017-003534-89-NL   France;Germany;Netherlands;United Kingdom;United States
      2017   Phase 1   EUCTR2017-003534-89-GB   France;Germany;Netherlands;United Kingdom
      -   Phase 3   EUCTR2018-003099-10-PL   Australia;Canada;France;Germany;Israel;Italy;Netherlands;New Zealand;Norway;Poland;Romania;Spain;United Kingdom;United States
      -   Phase 1   EUCTR2017-003534-89-DE   France;Germany;Netherlands;United Kingdom;United States
      -   Phase 1   EUCTR2017-003534-89-FR   France;Germany;Netherlands;United Kingdom;United States
   Dicerna Pharmaceuticals Inc . (a Novo Nordisk Company)
      2023   Phase 3   EUCTR2018-003099-10-NO   Australia;Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Netherlands;Norway;Poland;Romania;Spain;Türkiye;United Arab Emirates;United Kingdom;United States
   Dicerna Pharmaceuticals Inc.
      2021   Phase 2   EUCTR2021-001083-16-IT   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2020-002826-97-IT   Australia;France;Germany;Italy;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-IT   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Norway;Poland;Romania;Spain;United Kingdom;United States
   Dicerna Pharmaceuticals Inc. (a Novo Nordisk Company)
      2021   Phase 2   EUCTR2020-002826-97-DE   France;Germany;Italy;Lebanon;Morocco;Spain;United Arab Emirates;United Kingdom;United States
   Dicerna Pharmaceuticals, Inc.
      2020   Phase 1   EUCTR2020-000344-67-NL   France;Germany;Netherlands;United Kingdom;United States
      2020   Phase 1   EUCTR2020-000344-67-GB   France;Germany;Netherlands;United Kingdom;United States
      2020   Phase 1   EUCTR2020-000344-67-FR   France;Germany;Netherlands;United Kingdom;United States
      2020   Phase 1   EUCTR2020-000344-67-DE   France;Germany;Netherlands;United Kingdom;United States
   Dicerna Pharmaceuticals, Inc., Inc. (ICCC: intellim Corporation)
      2020   Phase 2   JPRN-jRCT2080225181   Asia except Japan;Japan;North America;Oceania
   Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
      2021   Phase 2   NCT04580420   France;Germany;Italy;Lebanon;Morocco;Romania;Spain;United Arab Emirates;United Kingdom;United States
      2020   Phase 1   NCT04555486   France;Germany;Netherlands;United Kingdom;United States
      2019   Phase 3   NCT04042402   Australia;Canada;France;Germany;Italy;Japan;Lebanon;Netherlands;Norway;Spain;Turkey;United Kingdom;United States
      2017   Phase 1   NCT03392896   France;Germany;Netherlands;United Kingdom;United States
   Novo Nordisk A/S
      2019   Phase 2   NCT03847909   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
Esomeprazole magnesium
   OxThera IP AB
      2009   -   EUCTR2009-015817-31-DE   Germany;Netherlands
Genetic analysis
   Mayo Clinic
      2003   -   NCT00589225   United States
Hematopoietic stem cell transplantation
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
Hydroxychloroquine
   The Hospital for Sick Children
      2019   Phase 2   NCT03856866   Canada
IMD preparative regimen
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States
Kidney imaging
   Biocodex
      2024   Phase 3   NCT06465472   -
Lumasiran
   ALNYLAM PHARMACEUTICALS, INC.
      2021   Phase 3   EUCTR2019-001346-17-IT   Australia;Belgium;Egypt;France;Germany;India;Israel;Italy;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
   Alnylam Pharmaceuticals
      2020   Phase 3   NCT04152200   Australia;Belgium;France;Israel;Italy;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
      2020   -   NCT04125472   Belgium;Canada;France;French Polynesia;Germany;Italy;Netherlands;Spain;Switzerland;United Kingdom;United States
      2019   Phase 3   NCT03905694   France;Germany;Israel;United Kingdom;United States
      2018   Phase 3   NCT03681184   France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States
      2018   Phase 2   NCT03350451   France;Germany;Israel;Netherlands;United Kingdom
      2016   Phase 1/Phase 2   NCT02706886   France;Germany;Israel;Jordan;Netherlands;United Kingdom;United States
   Alnylam Pharmaceuticals, Inc.
      2020   Phase 3   EUCTR2019-001346-17-GB   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
      2020   Phase 3   EUCTR2019-001346-17-FR   Australia;Belgium;Egypt;France;Germany;India;Israel;Italy;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
      2019   Phase 3   EUCTR2019-001346-17-NL   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
      2019   Phase 3   EUCTR2019-001346-17-BE   Australia;Belgium;Egypt;France;Germany;India;Israel;Italy;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
      2019   Phase 3   EUCTR2018-004014-17-GB   France;Germany;Israel;United Kingdom;United States
      2019   Phase 3   EUCTR2018-004014-17-FR   France;Germany;Israel;United Kingdom;United States
      2019   Phase 3   EUCTR2018-004014-17-DE   France;Germany;Israel;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001981-40-NL   France;Germany;Israel;Japan;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001981-40-GB   France;Germany;Israel;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States
      2018   Phase 3   EUCTR2018-001981-40-DE   France;Germany;Israel;Jordan;Netherlands;Switzerland;United Arab Emirates;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003134-24-GB   France;Germany;Israel;Netherlands;United Kingdom
      2017   Phase 2   EUCTR2016-003134-24-DE   France;Germany;Israel;Jordan;Netherlands;United Arab Emirates;United Kingdom;United States
      -   Phase 3   EUCTR2019-001346-17-DE   Australia;Belgium;Egypt;France;Germany;India;Israel;Jordan;Lebanon;Netherlands;Switzerland;Turkey;United Arab Emirates;United Kingdom;United States
Melphalan
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
Mycophenolate mofetil
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
Nedosiran
   Bowman Thomas
      2022   Phase 2   JPRN-jRCT2041220026   Canada;France;Germany;Italy;Japan;Lebanon;Poland;Spain;Turkey;United Kingdom;United States
   Dicerna Pharmaceuticals Inc
      2021   Phase 2   EUCTR2021-001083-16-PL   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2021-001083-16-ES   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2021-001083-16-DE   Canada;Germany;Italy;Japan;Lebanon;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2020-002826-97-FR   Australia;France;Germany;Italy;Japan;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-GB   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-PL   Australia;Canada;Egypt;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Saudi Arabia;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-GB   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003098-91-DE   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom;United States
   Dicerna Pharmaceuticals Inc . (a Novo Nordisk Company)
      2023   Phase 3   EUCTR2018-003099-10-NO   Australia;Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Netherlands;Norway;Poland;Romania;Spain;Türkiye;United Arab Emirates;United Kingdom;United States
   Dicerna Pharmaceuticals Inc.
      2021   Phase 2   EUCTR2021-001083-16-IT   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Kingdom;United States
      2021   Phase 2   EUCTR2020-002826-97-IT   Australia;France;Germany;Italy;Romania;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003099-10-IT   Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Norway;Poland;Romania;Spain;United Kingdom;United States
   Dicerna Pharmaceuticals Inc. (a Novo Nordisk Company)
      2021   Phase 2   EUCTR2020-002826-97-DE   France;Germany;Italy;Lebanon;Morocco;Spain;United Arab Emirates;United Kingdom;United States
   Dicerna Pharmaceuticals, Inc.
      2020   Phase 1   EUCTR2020-000344-67-NL   France;Germany;Netherlands;United Kingdom;United States
      2020   Phase 1   EUCTR2020-000344-67-GB   France;Germany;Netherlands;United Kingdom;United States
      2020   Phase 1   EUCTR2020-000344-67-FR   France;Germany;Netherlands;United Kingdom;United States
      2020   Phase 1   EUCTR2020-000344-67-DE   France;Germany;Netherlands;United Kingdom;United States
   Dicerna Pharmaceuticals, Inc., Inc. (ICCC: intellim Corporation)
      2020   Phase 2   JPRN-jRCT2080225181   Asia except Japan;Japan;North America;Oceania
   Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
      2025   -   NCT05993416   -
      2022   Phase 2   NCT05001269   Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Arab Emirates;United Kingdom;United States
Nexium 10 MG
   OxThera IP AB
      2009   -   EUCTR2009-015817-31-DE   Germany;Netherlands
OC3
   OxThera IP AB
      2009   -   EUCTR2009-015817-31-NL   Germany;Netherlands
      2009   -   EUCTR2009-015817-31-DE   Germany;Netherlands
   OxThera Inc.
      2008   Phase 3   EUCTR2007-002328-14-FR   France;Germany;Netherlands;United Kingdom
      2007   -   EUCTR2007-002328-14-NL   France;Germany;Netherlands;United Kingdom
      2007   Phase 3   EUCTR2007-002328-14-GB   France;Germany;Netherlands;United Kingdom
      2007   -   EUCTR2007-002328-14-DE   France;Germany;Netherlands;United Kingdom
   OxThera, Inc.
      2008   -   EUCTR2007-006187-30-NL   France;Germany;Netherlands;United Kingdom
      2008   Phase 3   EUCTR2007-006187-30-GB   France;Germany;Netherlands;United Kingdom
      2008   Phase 3   EUCTR2007-006187-30-FR   France;Germany;Netherlands;United Kingdom
      2008   -   EUCTR2007-006187-30-DE   France;Germany;Netherlands;United Kingdom
OC5
   OxThera AB
      2013   -   EUCTR2012-005606-22-GB   Germany;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2012-005606-22-DE   Germany;United Kingdom
   OxThera Intellectual Property AB
      2019   Phase 3   EUCTR2018-003576-12-GB   Belgium;Germany;Spain;Tunisia;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003576-12-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003576-12-DE   Belgium;Germany;Spain;Tunisia;United Kingdom;United States
      2018   Phase 3   EUCTR2017-000684-33-GB   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2018   Phase 3   EUCTR2017-000684-33-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2017-000684-33-BE   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-NL   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-FR   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-DE   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2014   Phase 2   EUCTR2013-004368-74-DE   Germany
      -   Phase 3   EUCTR2018-003576-12-BE   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
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
Oxabact (TM) - oxalobacter formigenes strain HC1
   OxThera, Inc.
      2008   -   EUCTR2007-006187-30-NL   France;Germany;Netherlands;United Kingdom
      2008   Phase 3   EUCTR2007-006187-30-GB   France;Germany;Netherlands;United Kingdom
      2008   Phase 3   EUCTR2007-006187-30-FR   France;Germany;Netherlands;United Kingdom
      2008   -   EUCTR2007-006187-30-DE   France;Germany;Netherlands;United Kingdom
Oxabact - oxalobacter formigenes, strain HC-1
   OxThera IP AB
      2009   -   EUCTR2009-015817-31-NL   Germany;Netherlands
      2009   -   EUCTR2009-015817-31-DE   Germany;Netherlands
Oxabact OC5 - oxalobacter formigenes HC-1
   OxThera
      2018   Phase 3   NCT03116685   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
Oxabact OC5 - oxalobacter formigenes strain HC-1
   OxThera
      2019   Phase 3   NCT03938272   Belgium;Germany;Spain;United Kingdom
Oxabact OC5 capsules
   OxThera
      2013   Phase 1/Phase 2   NCT02012985   France;Germany;United Kingdom
Oxabact(TM) - oxalobacter formigenes, strain HC1
   OxThera Inc.
      2008   Phase 3   EUCTR2007-002328-14-FR   France;Germany;Netherlands;United Kingdom
      2007   -   EUCTR2007-002328-14-NL   France;Germany;Netherlands;United Kingdom
      2007   Phase 3   EUCTR2007-002328-14-GB   France;Germany;Netherlands;United Kingdom
      2007   -   EUCTR2007-002328-14-DE   France;Germany;Netherlands;United Kingdom
Oxabact_oxalobacter formigenes strain HC-1
   OxThera Intellectual Property AB
      2019   Phase 3   EUCTR2018-003576-12-GB   Belgium;Germany;Spain;Tunisia;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003576-12-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003576-12-DE   Belgium;Germany;Spain;Tunisia;United Kingdom;United States
      2018   Phase 3   EUCTR2017-000684-33-GB   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2018   Phase 3   EUCTR2017-000684-33-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2017-000684-33-BE   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-NL   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-FR   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-DE   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      -   Phase 3   EUCTR2018-003576-12-BE   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
Oxabact_oxalobacter formigenes, strain HC-1
   OxThera AB
      2013   -   EUCTR2012-005606-22-GB   Germany;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2012-005606-22-DE   Germany;United Kingdom
   OxThera Intellectual Property AB
      2014   Phase 2   EUCTR2013-004368-74-DE   Germany
Oxabacter formigenes, strain HC1
   OxThera Intellectual Property AB
      2018   Phase 3   EUCTR2017-000684-33-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-NL   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
Oxalate decarboxylase
   Allena Pharmaceuticals, Inc.
      2018   Phase 2   EUCTR2017-003547-38-GB   Germany;United Kingdom;United States
      2018   Phase 2   EUCTR2017-003547-38-DE   Germany;United Kingdom;United States
Oxalobacter formigenes
   OxThera
      2014   Phase 2   NCT02000219   France;Germany;United States
      2009   Phase 2/Phase 3   NCT01037231   Germany;Netherlands;United States
      2007   Phase 2/Phase 3   NCT00638703   United States
Oxalobacter formigenes, strain HC 1
   OxThera AB
      2013   -   EUCTR2012-005606-22-GB   Germany;United Kingdom
      2013   Phase 1;Phase 2   EUCTR2012-005606-22-DE   Germany;United Kingdom
   OxThera Intellectual Property AB
      2019   Phase 3   EUCTR2018-003576-12-GB   Belgium;Germany;Spain;Tunisia;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003576-12-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003576-12-DE   Belgium;Germany;Spain;Tunisia;United Kingdom;United States
      2018   Phase 3   EUCTR2017-000684-33-GB   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2018   Phase 3   EUCTR2017-000684-33-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2018   Phase 3   EUCTR2017-000684-33-BE   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-NL   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-FR   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2017   Phase 3   EUCTR2017-000684-33-DE   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
      2014   Phase 2   EUCTR2013-004368-74-DE   Germany
      -   Phase 3   EUCTR2018-003576-12-BE   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
Oxalobacter formigenes, strain HC1
   OxThera, Inc.
      2008   -   EUCTR2007-006187-30-NL   France;Germany;Netherlands;United Kingdom
      2008   Phase 3   EUCTR2007-006187-30-GB   France;Germany;Netherlands;United Kingdom
      2008   Phase 3   EUCTR2007-006187-30-FR   France;Germany;Netherlands;United Kingdom
      2008   -   EUCTR2007-006187-30-DE   France;Germany;Netherlands;United Kingdom
Oxalobacterformigenes,strainhc1
   OxThera Intellectual Property AB
      2018   Phase 3   EUCTR2017-000684-33-GB   Belgium;France;Germany;Netherlands;Spain;Tunisia;United Kingdom;United States
Pyridoxine phosphate
   University of Cologne
      2010   -   EUCTR2009-015217-52-DE   Germany
Quality OF life questionnaires
   Biocodex
      2024   Phase 3   NCT06465472   -
Stem cell transplantation
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States
Stiripentol
   Biocodex
      2019   Phase 2   NCT03819647   France
Stiripentol oral capsule
   Biocodex
      2024   Phase 3   NCT06465472   -
Tacrolimus
   People's Hospital of Zhengzhou
      2013   -   ChiCTR-RO-14004471   China
Total body irradiation with marrow boosting
   Masonic Cancer Center, University of Minnesota
      2012   -   NCT01626092   United States
Urine samples collect
   Biocodex
      2024   Phase 3   NCT06465472   -
Ursodiol
   Children's Hospital Research Foundation University of Cincinnati
      1997   -   NCT00004442   -
Vitamin B 6
   University of Cologne
      2010   Phase 2   NCT01281878   Germany
YOLT-203
   Guangzhou Women and Children's Medical Center
      2025   Early Phase 1   NCT06892301   China
   RenJi Hospital
      2024   Early Phase 1   NCT06511349   China