240. フェニルケトン尿症 Disease details / Clinical trials / Drug dev / DR info /

5,6,7,8-tetrahydrobiopterin
   The Kennedy Institute-National Eye Clinic
      2005   Phase 2   NCT00260000   Denmark
A16ax07
   University Medical Center Groningen
      -   Phase 4   EUCTR2021-003777-63-NL   Netherlands
BH4
   ORPHANETICS Pharma Entwicklungs- GmbH
      2006   -   EUCTR2006-000648-15-AT   Austria
   Orphanetics Pharma Entwicklungs GmbH
      2007   Phase 2/Phase 3   NCT00432822   -
   Taipei Veterans General Hospital, Taiwan
      2020   Phase 4   NCT04227080   -
   The University of Texas Medical Branch, Galveston
      2005   Phase 1   NCT00244218   United States
Blood samples
   University Hospital, Tours
      2021   -   NCT04879277   France
BMN 165
   BioMarin Pharmaceutical
      2013   Phase 3   NCT01819727   United States
      2012   Phase 2   NCT01560286   United States
   BioMarin Pharmaceutical Inc.
      -   Phase 3   EUCTR2021-005058-27-DE   Germany;United States
   BioMarin Pharmaceutical Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-jRCT2080224573   Japan
BMN 307
   BioMarin Pharmaceutical
      2020   Phase 1/Phase 2   NCT04480567   United Kingdom;United States
   BioMarin Pharmaceutical Inc.
      2019   Phase 1;Phase 2   EUCTR2019-001878-28-GB   Australia;Germany;Italy;Spain;Taiwan;Turkey;United Kingdom;United States
BMN165 20MG/DAY
   BioMarin Pharmaceutical
      2013   Phase 3   NCT01889862   United States
BMN165 40MG/DAY
   BioMarin Pharmaceutical
      2013   Phase 3   NCT01889862   United States
CBT102-A capsule
   Children's Hospital of Fudan University
      2023   -   NCT05948020   China
CDX 6114
   Nestlé
      2019   Phase 1   NCT04085666   Australia;Germany
CNSA-001
   PTC Therapeutics
      2018   Phase 1/Phase 2   NCT03519711   Germany;United States
Cohort 1 0.225G
   Nestlé
      2020   Phase 1   NCT04256655   -
Cohort 2 0.75G
   Nestlé
      2020   Phase 1   NCT04256655   -
Cohort 3 2.25 G
   Nestlé
      2020   Phase 1   NCT04256655   -
Corn/SOY OIL
   Emory University
      2007   -   NCT00892554   United States
Diet treatment
   University Hospitals Cleveland Medical Center
      2018   Phase 4   NCT02677870   United States
Dietary supplement FOR PKU patients
   University Hospital, Tours
      2019   -   NCT03924180   France
Docosahexaenoic acid
   Emory University
      2007   -   NCT00892554   United States
Free amino acids intake
   University of British Columbia
      2019   -   NCT03939052   Canada
Glycomacropeptide-based protein substitute
   Nutricia UK Ltd
      2016   -   NCT02915510   United Kingdom
Glytactin
   Ajinomoto Co., Inc.
      2023   -   NCT06332105   United Kingdom
GMP intervention product
   Nutricia UK Ltd
      2025   -   NCT06941532   -
Hepatocyte transplant
   Ira Fox
      2011   Phase 1/Phase 2   NCT01465100   United States
HMI-102
   Homology Medicines, Inc
      2019   Phase 1/Phase 2   NCT03952156   United States
HMI-103
   Homology Medicines, Inc
      2022   Phase 1   NCT05222178   United States
Immunosuppression
   Ira Fox
      2011   Phase 1/Phase 2   NCT01465100   United States
Iron supplementation
   ASST Santi Paolo e Carlo
      2020   Phase 2   NCT06792240   Italy
JNT-517 suspension
   Jnana Therapeutics
      2022   Phase 1   NCT05781399   Australia;United States
JNT-517 tablet
   Jnana Therapeutics
      2025   Phase 3   NCT06971731   -
      2025   Phase 2   NCT06637514   -
      2025   Phase 3   NCT06628128   -
      2022   Phase 1   NCT05781399   Australia;United States
Kuvan
   BioMarin International Ltd
      2011   Phase 3   EUCTR2009-015768-33-SK   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-DE   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-BE   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-AT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2010   Phase 3   EUCTR2009-015768-33-CZ   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   BioMarin International Ltd.
      2013   Phase 4   EUCTR2009-015844-41-GB   Germany;Italy;Spain;United Kingdom
      2013   Phase 4   EUCTR2009-015844-41-DE   Germany;Italy;Spain;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-GB   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   BioMarin Pharmaceutical
      2017   -   NCT03864029   China
      2013   Phase 4   NCT01965912   Germany;Italy;Spain;United Kingdom
      2011   Phase 3   NCT01376908   Austria;Belgium;Czech Republic;Czechia;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2008   -   NCT00778206   Puerto Rico;United States
   Dr. Linda Randolph
      2013   -   NCT01806051   United States
   Emory University
      2011   Phase 2   NCT01395394   United States
   Graz Medical University
      2010   -   EUCTR2010-019767-11-AT   Austria
   MERCK SERONO SA
      2011   Phase 3   EUCTR2009-015768-33-IT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   Merck KGaA
      2014   -   EUCTR2010-024311-13-NL   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
      2014   -   EUCTR2010-024311-13-DE   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
      2013   -   EUCTR2010-024311-13-IT   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
      2013   -   EUCTR2010-024311-13-ES   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
      2013   -   EUCTR2010-024311-13-BE   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
      2013   Phase 4   EUCTR2009-015844-41-IT   Germany;Italy;Spain;United Kingdom
      2013   Phase 4   EUCTR2009-015844-41-ES   Germany;Italy;Spain;United Kingdom
      2012   Phase 3   NCT01732471   Germany
      2010   Phase 4   NCT01082328   Norway
   Merck Serono Norway
      2010   -   EUCTR2009-018168-81-DK   Denmark
      -   Phase 4   EUCTR2009-018168-81-NO   Denmark;Norway
   Merck Serono S.A. - Geneva
      2011   Phase 3   EUCTR2009-015768-33-PT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-NL   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   Merck Serono, an affiliate of E. Merck AB
      -   Phase 4   EUCTR2009-012978-12-NO   Norway
   University Medical Center Groningen
      2014   Phase 4   EUCTR2010-021343-41-NL   Netherlands
      -   Phase 4   EUCTR2021-003777-63-NL   Netherlands
   University of Miami
      2008   Phase 2   NCT00841100   United States
   University of Southern California
      2012   Phase 4   NCT01617070   United States
   Washington University School of Medicine
      2009   -   NCT00827762   United States
      2008   -   NCT00730080   United States
Kuvan 100MG soluble tablet
   Merck Serono Middle East FZ-LLC
      -   -   EUCTR2015-001650-15-Outside-EU/EEA   Russian Federation;Ukraine
Kuvantm therapy
   Emory University
      2008   -   NCT00688844   United States
L-AA
   Ajinomoto Co., Inc.
      2023   -   NCT06332105   United Kingdom
L-carnitine
   Mansoura University
      2025   Phase 4   NCT06901323   Egypt
Large neutral amino acid therapy
   University of Southern California
      2012   Phase 4   NCT01617070   United States
Liver biopsy
   Ira Fox
      2011   Phase 1/Phase 2   NCT01465100   United States
Liver evaluation
   Ira Fox
      2011   Phase 1/Phase 2   NCT01465100   United States
LOW calorie protein substitute
   Nutricia UK Ltd
      2023   -   NCT04272736   -
Meal challenge
   Emory University
      2011   Phase 2   NCT01395394   United States
Medikinet
   Amsterdam UMC
      2021   Phase 4   EUCTR2021-001174-29-NL   Netherlands
Moxifloxacin
   BioMarin Pharmaceutical
      2008   Phase 1   NCT00789568   United States
MRNA-3210
   ModernaTX, Inc.
      2024   Phase 1/Phase 2   NCT06147856   Australia;United States
NAP
   BioMarin International Ltd
      2011   Phase 3   EUCTR2009-015768-33-SK   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-DE   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-BE   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-AT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2010   Phase 3   EUCTR2009-015768-33-CZ   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   BioMarin International Ltd.
      2011   Phase 3   EUCTR2009-015768-33-GB   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   Merck Serono S.A. - Geneva
      2011   Phase 3   EUCTR2009-015768-33-PT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-NL   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
Neuro-psychological assessment
   Ira Fox
      2011   Phase 1/Phase 2   NCT01465100   United States
NGGT002
   First Affiliated Hospital Bengbu Medical College
      2023   Early Phase 1   NCT06061614   China
   NGGT (Suzhou) Biotechnology Co., Ltd.
      2024   Phase 1/Phase 2   NCT06687733   China
   NGGT INC.
      2025   Phase 1/Phase 2   NCT06332807   United States
Normal (control) diet
   University of Glasgow
      2014   -   NCT02440932   United Kingdom
Palynziq
   BioMarin Pharmaceutical Inc.
      -   Phase 3   EUCTR2021-005058-27-DE   Germany;United States
   Emory University
      2019   -   NCT04404530   United States
Pegvaliase
   BioMarin Pharmaceutical
      2024   -   NCT06305234   United States
      2022   -   NCT05813678   Germany;Italy;United States
      2022   -   NCT05579548   Germany;Italy;United States
      2022   Phase 3   NCT05270837   Germany;United States
      2018   Phase 3   NCT03694353   United States
   BioMarin Pharmaceutical Inc.
      2019   Phase 3   EUCTR2018-000648-25-FR   Austria;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey
      2019   Phase 3   EUCTR2018-000648-25-DE   Austria;France;Germany;Italy;Netherlands;Spain;Turkey
      -   Phase 3   EUCTR2021-005058-27-DE   Germany;United States
      -   Phase 3   EUCTR2018-000648-25-NL   Austria;France;Germany;Italy;Netherlands;Spain;Turkey
   BioMarin Pharmaceutical Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-jRCT2080224573   Japan
   Boston Children's Hospital
      2019   -   NCT03792451   United States
Pegvaliase-pqpz
   University of Missouri-Columbia
      2022   -   NCT05356377   United States
Phenoptin
   BIOMARIN
      2006   -   EUCTR2006-000839-10-IT   Germany;Ireland;Italy;Spain;United Kingdom
      2005   -   EUCTR2004-004513-41-IT   Germany;Ireland;Italy;United Kingdom
      2005   -   EUCTR2004-004512-23-IT   Germany;Ireland;Italy;United Kingdom
      2005   -   EUCTR2004-002071-16-IT   Germany;Italy;United Kingdom
   BioMarin Pharmaceutical
      2006   Phase 2   NCT00355264   Germany;United States
   BioMarin Pharmaceutical Inc
      2006   -   EUCTR2005-003778-13-DE   Germany
   BioMarin Pharmaceutical Inc.
      2006   -   EUCTR2006-000839-10-IE   Germany;Ireland;Italy;Spain;United Kingdom
      2006   Phase 3   EUCTR2006-000839-10-GB   Germany;Ireland;Italy;Spain;United Kingdom
      2006   -   EUCTR2006-000839-10-ES   Germany;Ireland;Italy;Spain;United Kingdom
      2006   -   EUCTR2006-000839-10-DE   Germany;Ireland;Italy;Spain;United Kingdom
      2006   Phase 3   EUCTR2005-003777-24-GB   Germany;Spain;United Kingdom
      2006   Phase 3   EUCTR2005-003777-24-ES   Germany;Spain;United Kingdom
      2006   -   EUCTR2005-003777-24-DE   Germany;Spain
      2005   Phase 3   EUCTR2004-004513-41-IE   Germany;Ireland;Italy;United Kingdom
      2005   Phase 3   EUCTR2004-004513-41-GB   Germany;Ireland;Italy;United Kingdom
      2005   -   EUCTR2004-004513-41-DE   Germany;Ireland;Italy;United Kingdom
      2005   Phase 3   EUCTR2004-004512-23-IE   Germany;Ireland;Italy;United Kingdom
      2005   Phase 3   EUCTR2004-004512-23-GB   Germany;Ireland;Italy;United Kingdom
      2005   Phase 2   EUCTR2004-002071-16-GB   Germany;Ireland;Italy;United Kingdom
      2005   -   EUCTR2004-002071-16-DE   Germany;Italy;United Kingdom
      -   -   EUCTR2004-004512-23-DE   Germany;Ireland;Italy;United Kingdom
      -   Phase 2   EUCTR2004-002071-16-IE   Germany;Ireland;Italy;United Kingdom
Phenylalanine
   University Hospital Inselspital, Berne
      2019   Phase 4   NCT03788343   Switzerland
   metaX Institut fuer Diatetik GmbH
      2021   -   NCT04375592   United Kingdom
Phenylalanine (PHE)-restricted diet
   BioMarin Pharmaceutical
      2011   Phase 3   NCT01376908   Austria;Belgium;Czech Republic;Czechia;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
Phenylalanine restricted diet
   Assiut University
      2020   -   NCT04302194   -
   National Center for Research Resources (NCRR)
      1983   -   NCT00006142   -
Phenylalanine-free milk
   Chang Gung Memorial Hospital
      2021   -   NCT04896281   Taiwan
Phenylalanine-free protein substitute tablets
   Nutricia UK Ltd
      2019   -   NCT03333720   -
Phenylketonuria-type diet
   University of Glasgow
      2014   -   NCT02440932   United Kingdom
PKU easy microtabs plus
   Galen Limited
      2025   -   NCT06694662   United Kingdom
PKU explore
   Vitaflo International, Ltd
      2022   -   NCT04898829   France
      2017   -   NCT03168399   United Kingdom
PKU golike
   APR Applied Pharma Research s.a.
      2023   -   NCT05827536   Italy
PKU sphere
   Vitaflo International, Ltd
      2020   -   NCT04679467   Italy
      2019   -   NCT04224142   United Kingdom
      2018   -   NCT03771391   Germany
      2018   -   NCT03419819   United States
PKU sphere liquid
   Vitaflo International, Ltd
      2021   -   NCT05096988   United Kingdom
PKU start
   Vitaflo International, Ltd
      2017   -   NCT03058848   United Kingdom
PKU UP
   Vitaflo International, Ltd
      2024   -   NCT05995717   United Kingdom
PKU/TYR GMP protein substitute
   Nutricia UK Ltd
      2020   -   NCT05062226   United Kingdom
Preparative radiation therapy
   Ira Fox
      2011   Phase 1/Phase 2   NCT01465100   United States
Protein intake
   University of British Columbia
      2013   -   NCT01965691   Canada
PTC923
   PTC THERAPEUTICS INC.
      2022   Phase 3   EUCTR2021-000497-28-IT   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-000474-29-IT   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
   PTC Therapeutics
      2022   Phase 3   NCT05166161   Australia;Brazil;Canada;Czechia;Denmark;France;Georgia;Germany;Italy;Japan;Mexico;Netherlands;Poland;Portugal;Slovenia;Spain;Turkey;United Kingdom;United States
      2021   Phase 3   NCT05099640   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
   PTC Therapeutics, Inc.
      2023   Phase 3   EUCTR2021-000497-28-NL   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-000497-28-PT   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-000497-28-DK   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-000474-29-PT   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-000474-29-NL   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-000474-29-ES   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-000474-29-DK   Australia;Brazil;Canada;Denmark;France;Germany;Italy;Mexico;Netherlands;Portugal;Spain;United States
      -   Phase 3   EUCTR2021-000497-28-DE   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2021-000474-29-DE   Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom;United States
Ravpal-PEG
   BioMarin Pharmaceutical
      2012   Phase 2   NCT01560286   United States
      2011   Phase 2   NCT01212744   United States
      2009   Phase 2   NCT00924703   United States
      2008   Phase 1   NCT00634660   United States
   BioMarin Pharmaceutical Inc.
      2019   Phase 3   EUCTR2018-000648-25-FR   Austria;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey
      2019   Phase 3   EUCTR2018-000648-25-DE   Austria;France;Germany;Italy;Netherlands;Spain;Turkey
      -   Phase 3   EUCTR2021-005058-27-DE   Germany;United States
      -   Phase 3   EUCTR2018-000648-25-NL   Austria;France;Germany;Italy;Netherlands;Spain;Turkey
Ravpal-PEG 0.001 MG/KG
   BioMarin Pharmaceutical
      2009   Phase 2   NCT00925054   United States
Ravpal-PEG 0.003 MG/KG
   BioMarin Pharmaceutical
      2009   Phase 2   NCT00925054   United States
Ravpal-PEG 0.01 MG/KG
   BioMarin Pharmaceutical
      2009   Phase 2   NCT00925054   United States
Ravpal-PEG 0.03 MG/KG
   BioMarin Pharmaceutical
      2009   Phase 2   NCT00925054   United States
Ravpal-PEG 0.1 MG/KG
   BioMarin Pharmaceutical
      2009   Phase 2   NCT00925054   United States
RDD TO palynziq
   BioMarin Pharmaceutical
      2025   Phase 4   NCT06780332   United States
Restricted phenylalanine diet
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      1984   -   NCT00065299   United States
Routine
   Nutricia UK Ltd
      2017   -   NCT03167697   United Kingdom
RTX-134
   Rubius Therapeutics
      2020   Phase 1   NCT04110496   United States
Sapropterin
   BIOMARIN
      2006   -   EUCTR2006-000839-10-IT   Germany;Ireland;Italy;Spain;United Kingdom
   BioMarin International Ltd.
      2013   Phase 4   EUCTR2009-015844-41-GB   Germany;Italy;Spain;United Kingdom
      2013   Phase 4   EUCTR2009-015844-41-DE   Germany;Italy;Spain;United Kingdom
   BioMarin Pharmaceutical
      2014   Phase 2   NCT01977820   Belgium;Germany;Italy;Netherlands;Spain;Switzerland
   BioMarin Pharmaceutical Inc
      2006   -   EUCTR2005-003778-13-DE   Germany
   Children's Hospital of Philadelphia
      2010   -   NCT00986973   United States
   MERCK SERONO SA
      2011   Phase 3   EUCTR2009-015768-33-IT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   Merck KGaA
      2013   Phase 4   EUCTR2009-015844-41-IT   Germany;Italy;Spain;United Kingdom
      2013   Phase 4   EUCTR2009-015844-41-ES   Germany;Italy;Spain;United Kingdom
   ORPHANETICS Pharma Entwicklungs- GmbH
      2006   -   EUCTR2006-000648-15-AT   Austria
   The University of Texas Health Science Center, Houston
      2011   -   NCT01541397   United States
   Universidade do Porto
      2019   -   NCT03820804   Portugal
   University of Missouri-Columbia
      2009   -   NCT00964236   United States
   Washington University School of Medicine
      2008   -   NCT00730080   United States
Sapropterin dihydrochloride
   BioMarin Pharmaceutical
      2010   Phase 3   NCT01114737   Canada;United States
      2009   Phase 3   NCT00838435   Canada;United States
      2008   Phase 1   NCT00789568   United States
      2007   -   NCT00484991   United States
      2006   Phase 3   NCT00332189   United States
      2005   Phase 3   NCT00225615   United States
      2004   Phase 2   NCT00104260   United States
   BioMarin Pharmaceutical Inc.
      2006   -   EUCTR2006-000839-10-IE   Germany;Ireland;Italy;Spain;United Kingdom
      2006   Phase 3   EUCTR2006-000839-10-GB   Germany;Ireland;Italy;Spain;United Kingdom
      2006   -   EUCTR2006-000839-10-ES   Germany;Ireland;Italy;Spain;United Kingdom
      2006   -   EUCTR2006-000839-10-DE   Germany;Ireland;Italy;Spain;United Kingdom
      2006   Phase 3   EUCTR2005-003777-24-GB   Germany;Spain;United Kingdom
      2006   Phase 3   EUCTR2005-003777-24-ES   Germany;Spain;United Kingdom
      2006   -   EUCTR2005-003777-24-DE   Germany;Spain
   Children's Research Institute
      2011   -   NCT01412437   United States
   Merck KGaA
      2014   -   EUCTR2010-024311-13-NL   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
      2014   -   EUCTR2010-024311-13-DE   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
      2013   -   EUCTR2010-024311-13-IT   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
      2013   -   EUCTR2010-024311-13-ES   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
      2013   -   EUCTR2010-024311-13-BE   Belgium;France;Germany;Italy;Netherlands;Spain;Switzerland
   Merck Serono S.A. - Geneva
      2011   Phase 3   EUCTR2009-015768-33-NL   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   Merck Serono, an affiliate of E. Merck AB
      -   Phase 4   EUCTR2009-012978-12-NO   Norway
   Tulane University School of Medicine
      2011   -   NCT01274026   United States
   University Medical Center Groningen
      -   Phase 4   EUCTR2021-003777-63-NL   Netherlands
Sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
   BioMarin Pharmaceutical
      2005   Phase 3   NCT00104247   United States
Sapropterin dihydrocholoride
   BioMarin International Ltd
      2011   Phase 3   EUCTR2009-015768-33-SK   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-DE   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-BE   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-AT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2010   Phase 3   EUCTR2009-015768-33-CZ   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   BioMarin International Ltd.
      2011   Phase 3   EUCTR2009-015768-33-GB   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   Merck Serono Middle East FZ-LLC
      -   -   EUCTR2015-001650-15-Outside-EU/EEA   Russian Federation;Ukraine
   Merck Serono S.A. - Geneva
      2011   Phase 3   EUCTR2009-015768-33-PT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
Sapropterin hydrochloride
   BIOMARIN
      2005   -   EUCTR2004-002071-16-IT   Germany;Italy;United Kingdom
   Merck Serono Norway
      2010   -   EUCTR2009-018168-81-DK   Denmark
      -   Phase 4   EUCTR2009-018168-81-NO   Denmark;Norway
Sapropterin, sapropterin dihydrochloride
   BioMarin Pharmaceutical Inc.
      2005   Phase 3   EUCTR2004-004513-41-GB   Germany;Ireland;Italy;United Kingdom
      2005   -   EUCTR2004-004513-41-DE   Germany;Ireland;Italy;United Kingdom
Sapropterin, sapropterin hydrochloride
   BioMarin Pharmaceutical Inc.
      2005   Phase 3   EUCTR2004-004513-41-IE   Germany;Ireland;Italy;United Kingdom
      2005   Phase 3   EUCTR2004-004512-23-IE   Germany;Ireland;Italy;United Kingdom
      2005   Phase 3   EUCTR2004-004512-23-GB   Germany;Ireland;Italy;United Kingdom
      2005   Phase 2   EUCTR2004-002071-16-GB   Germany;Ireland;Italy;United Kingdom
      2005   -   EUCTR2004-002071-16-DE   Germany;Italy;United Kingdom
      -   -   EUCTR2004-004512-23-DE   Germany;Ireland;Italy;United Kingdom
      -   Phase 2   EUCTR2004-002071-16-IE   Germany;Ireland;Italy;United Kingdom
Sapropterindihydrochloride
   Merck Serono Norway
      2010   -   EUCTR2009-018168-81-DK   Denmark
      -   Phase 4   EUCTR2009-018168-81-NO   Denmark;Norway
Saproterin dihydrochloride
   BioMarin Pharmaceutical Inc
      2006   -   EUCTR2005-003778-13-DE   Germany
   University Hospitals Cleveland Medical Center
      2018   Phase 4   NCT02677870   United States
SAR444836
   Sanofi
      2023   Phase 1/Phase 2   NCT05972629   Argentina;Brazil;Germany;Israel;Turkey;United States
Sepiapterin
   PTC Therapeutics
      2024   Phase 3   NCT06302348   United States
SYNB1618
   Synlogic
      2020   Phase 2   NCT04534842   United States
      2018   Phase 1/Phase 2   NCT03516487   United States
SYNB1934
   Synlogic
      2020   Phase 2   NCT04534842   United States
SYNB1934v1
   Synlogic
      2023   Phase 3   NCT05764239   Canada;Georgia;Turkey;United States
Synergy
   Nutricia UK Ltd
      2017   -   NCT03167697   United Kingdom
T1401
   BIOMARIN
      2006   -   EUCTR2006-000839-10-IT   Germany;Ireland;Italy;Spain;United Kingdom
   BioMarin Pharmaceutical Inc
      2006   -   EUCTR2005-003778-13-DE   Germany
   BioMarin Pharmaceutical Inc.
      2006   -   EUCTR2006-000839-10-IE   Germany;Ireland;Italy;Spain;United Kingdom
      2006   Phase 3   EUCTR2006-000839-10-GB   Germany;Ireland;Italy;Spain;United Kingdom
      2006   -   EUCTR2006-000839-10-ES   Germany;Ireland;Italy;Spain;United Kingdom
      2006   -   EUCTR2006-000839-10-DE   Germany;Ireland;Italy;Spain;United Kingdom
      2006   Phase 3   EUCTR2005-003777-24-GB   Germany;Spain;United Kingdom
      2006   Phase 3   EUCTR2005-003777-24-ES   Germany;Spain;United Kingdom
      2006   -   EUCTR2005-003777-24-DE   Germany;Spain
      2005   Phase 3   EUCTR2004-004513-41-IE   Germany;Ireland;Italy;United Kingdom
      2005   Phase 3   EUCTR2004-004513-41-GB   Germany;Ireland;Italy;United Kingdom
      2005   -   EUCTR2004-004513-41-DE   Germany;Ireland;Italy;United Kingdom
      2005   Phase 3   EUCTR2004-004512-23-IE   Germany;Ireland;Italy;United Kingdom
      2005   Phase 3   EUCTR2004-004512-23-GB   Germany;Ireland;Italy;United Kingdom
      2005   Phase 2   EUCTR2004-002071-16-GB   Germany;Ireland;Italy;United Kingdom
      2005   -   EUCTR2004-002071-16-DE   Germany;Italy;United Kingdom
      -   -   EUCTR2004-004512-23-DE   Germany;Ireland;Italy;United Kingdom
      -   Phase 2   EUCTR2004-002071-16-IE   Germany;Ireland;Italy;United Kingdom
Tetrahydrobiopterin
   BioMarin International Ltd
      2011   Phase 3   EUCTR2009-015768-33-SK   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-DE   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-BE   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-AT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2010   Phase 3   EUCTR2009-015768-33-CZ   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   BioMarin International Ltd.
      2013   Phase 4   EUCTR2009-015844-41-GB   Germany;Italy;Spain;United Kingdom
      2013   Phase 4   EUCTR2009-015844-41-DE   Germany;Italy;Spain;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-GB   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   John F. Kennedy Institute
      2004   -   EUCTR2004-002365-21-DK   Denmark
   Merck KGaA
      2013   Phase 4   EUCTR2009-015844-41-IT   Germany;Italy;Spain;United Kingdom
      2013   Phase 4   EUCTR2009-015844-41-ES   Germany;Italy;Spain;United Kingdom
   Merck Serono S.A. - Geneva
      2011   Phase 3   EUCTR2009-015768-33-PT   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
      2011   Phase 3   EUCTR2009-015768-33-NL   Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom
   ORPHANETICS Pharma Entwicklungs- GmbH
      2006   -   EUCTR2006-000648-15-AT   Austria
   Orphanetics Pharma Entwicklungs GmbH
      2007   Phase 2/Phase 3   NCT00432822   -
   The University of Texas Medical Branch, Galveston
      2005   Phase 1   NCT00244218   United States
   University of Massachusetts, Amherst
      2021   Phase 1   NCT04014712   United States
Whole body PHE oxidation testing
   Ira Fox
      2011   Phase 1/Phase 2   NCT01465100   United States