240. フェニルケトン尿症 Disease details / Clinical trials / Drug dev / DR info /
臨床試験数 : 143 / 薬物数 : 90 - (DrugBank : 10) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
薬物ごとの開発者（Primary Sponsor）、臨床試験情報（抜粋）です。
(S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
PTC Therapeutics, Inc.
2022 Phase 3 EUCTR2021-000474-29-PT 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;
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;
Taipei Veterans General Hospital, Taiwan
2020 Phase 4 NCT04227080 -
BMN 165
BioMarin Pharmaceutical
2013 Phase 3 NCT01819727 United States;
BioMarin Pharmaceutical Inc./CMIC Co., Ltd.
2019 Phase 3 JPRN-JapicCTI-194642 Japan;
BMN 165 (rAvPAL-PEG)
BioMarin Pharmaceutical
2012 Phase 2 NCT01560286 United States;
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;
Blood samples
University Hospital, Tours
2021 - NCT04879277 France;
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
Children's Research Institute
2011 - NCT01412437 United States;
Dietary Supplement for PKU patients
University Hospital, Tours
2019 - NCT03924180 France;
Free amino acids intake
University of British Columbia
2019 - NCT03939052 Canada;
Glycomacropeptide-based protein substitute
Nutricia UK Ltd
2016 - NCT02915510 United Kingdom;
HMI-103
Homology Medicines, Inc
2022 Phase 1 NCT05222178 United States;
Hepatocyte Transplant
Ira Fox
2022 Phase 1/Phase 2 NCT01465100 United States;
High oleic sunflower oil
Ludwig-Maximilians - University of Munich
2009 - NCT00909012 Germany;Italy;Spain;United Kingdom;
Immunosuppression
Ira Fox
2022 Phase 1/Phase 2 NCT01465100 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
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 KGaA
2013 Phase 4 EUCTR2009-015844-41-IT Germany;Italy;Spain;United Kingdom;
2013 - EUCTR2009-015844-41-ES Germany;Spain;United Kingdom;
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-NL Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
2011 - EUCTR2009-015768-33-PT Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
University Medical Center Groningen
2014 Phase 4 EUCTR2010-021343-41-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;
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;
Kuvan®
BioMarin Pharmaceutical
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;
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;
2012 Phase 3 NCT01732471 Germany;
2010 Phase 4 NCT01082328 Norway;
Kuvan® (sapropterin dihydrochloride)
Merck Serono, an affiliate of E. Merck AB
- Phase 4 EUCTR2009-012978-12-NO Norway;
University Medical Center Groningen
- Phase 4 EUCTR2021-003777-63-NL Netherlands;
Large Neutral Amino Acid Therapy
University of Southern California
2012 Phase 4 NCT01617070 United States;
Liver Biopsy
Ira Fox
2022 Phase 1/Phase 2 NCT01465100 United States;
Low calorie protein substitute
Nutricia UK Ltd
2020 - NCT04272736 -
Medikinet
Amsterdam UMC
2021 Phase 4 EUCTR2021-001174-29-NL Netherlands;
Microalgal oil
Ludwig-Maximilians - University of Munich
2009 - NCT00909012 Germany;Italy;Spain;United Kingdom;
Moxifloxacin
BioMarin Pharmaceutical
2008 Phase 1 NCT00789568 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-NL Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
2011 - EUCTR2009-015768-33-PT Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
PKU Explore
Vitaflo International, Ltd
2017 - NCT03168399 United Kingdom;
PKU Sphere
Vitaflo International, Ltd
2018 - NCT03771391 Germany;
2018 - NCT03419819 United States;
PKU Start
Vitaflo International, Ltd
2017 - NCT03058848 United Kingdom;
PKU explore
Vitaflo International, Ltd
2021 - NCT04898829 France;
PKU express plus
Vitaflo International, Ltd
2021 - NCT05051657 United Kingdom;
PKU sphere
Vitaflo International, Ltd
2020 - NCT04679467 Italy;
2019 - NCT04224142 United Kingdom;
PKU sphere liquid
Vitaflo International, Ltd
2021 - NCT05096988 United Kingdom;
PKU/TYR GMP Protein Substitute
Nutricia UK Ltd
2020 - NCT05062226 United Kingdom;
PTC923
PTC Therapeutics
2022 Phase 3 NCT05166161 Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;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.
2022 Phase 3 EUCTR2021-000474-29-PT 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;
Palynziq
Emory University
2019 - NCT04404530 United States;
Pegvaliase
BioMarin Pharmaceutical
2018 Phase 3 NCT03694353 United States;
BioMarin Pharmaceutical Inc./CMIC Co., Ltd.
2019 Phase 3 JPRN-JapicCTI-194642 Japan;
Boston Children's Hospital
2019 - NCT03792451 United States;
Phe
BioMarin Pharmaceutical
2011 Phase 3 NCT01376908 Austria;Belgium;Czech Republic;Czechia;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
Ira Fox
2022 Phase 1/Phase 2 NCT01465100 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 Phase 3 EUCTR2006-000839-10-GB Germany;Ireland;Italy;Spain;United Kingdom;
2006 Phase 3 EUCTR2005-003777-24-GB Germany;Spain;United Kingdom;
2006 - EUCTR2006-000839-10-IE 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 - EUCTR2005-003777-24-ES Germany;Spain;
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 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 - EUCTR2004-004513-41-DE Germany;Ireland;Italy;United Kingdom;
2005 - EUCTR2004-002071-16-DE Germany;Italy;United Kingdom;
- Phase 2 EUCTR2004-002071-16-IE Germany;Ireland;Italy;United Kingdom;
- - EUCTR2004-004512-23-DE Germany;Ireland;Italy;United Kingdom;
Phenylalanine
BioMarin Pharmaceutical
2011 Phase 3 NCT01376908 Austria;Belgium;Czech Republic;Czechia;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
1984 - NCT00065299 United States;
National Center for Research Resources (NCRR)
1983 - NCT00006142 -
University Hospital Inselspital, Berne
2019 Phase 4 NCT03788343 Switzerland;
Phenylalanine restricted diet
Assiut University
2020 - NCT04302194 -
Phenylalanine-free protein substitute in tablet form (XPhe minis)
metaX Institut fuer Diatetik GmbH
2021 - NCT04375592 United Kingdom;
Phenylalanine-free protein substitute tablets
Nutricia UK Ltd
2019 - NCT03333720 -
Phenylketonuria-type diet
University of Glasgow
2014 - NCT02440932 United Kingdom;
Protein intake
University of British Columbia
2013 - NCT01965691 Canada;
RAvPAL-PEG
BioMarin Pharmaceutical
2011 Phase 2 NCT01212744 United States;
2009 Phase 2 NCT00925054 United States;
2009 Phase 2 NCT00924703 United States;
2008 Phase 1 NCT00634660 United States;
RTX-134
Rubius Therapeutics
2020 Phase 1 NCT04110496 United States;
SAPROPTERIN DIHYDROCHLORIDE
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;
SAPROPTERIN HYDROCHLORIDE
Merck Serono Norway
2010 - EUCTR2009-018168-81-DK Denmark;
- Phase 4 EUCTR2009-018168-81-NO Denmark;Norway;
SYNB1618
Synlogic
2020 Phase 2 NCT04534842 United States;
2018 Phase 1/Phase 2 NCT03516487 United States;
SYNB1934
Synlogic
2020 Phase 2 NCT04534842 United States;
Sapropterin
BIOMARIN
2006 - EUCTR2006-000839-10-IT Germany;Ireland;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;
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;
Sapropterin (Kuvan)
Washington University School of Medicine
2008 - NCT00730080 United States;
Sapropterin Dihydrochloride
Merck Serono S.A. - Geneva
2011 Phase 3 EUCTR2009-015768-33-NL Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
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 - EUCTR2009-015768-33-PT Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
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 Phase 3 EUCTR2006-000839-10-GB Germany;Ireland;Italy;Spain;United Kingdom;
2006 Phase 3 EUCTR2005-003777-24-GB Germany;Spain;United Kingdom;
2006 - EUCTR2006-000839-10-IE 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 - EUCTR2005-003777-24-ES Germany;Spain;
2006 - EUCTR2005-003777-24-DE Germany;Spain;
Children's Research Institute
2011 - NCT01412437 United States;
Tulane University School of Medicine
2011 - NCT01274026 United States;
Sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
BioMarin Pharmaceutical
2005 Phase 3 NCT00104247 United States;
Sapropterin hydrochloride
BIOMARIN
2005 - EUCTR2004-002071-16-IT Germany;Italy;United Kingdom;
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;
- Phase 2 EUCTR2004-002071-16-IE Germany;Ireland;Italy;United Kingdom;
- - EUCTR2004-004512-23-DE 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;
Synergy
Nutricia UK Ltd
2017 - NCT03167697 United Kingdom;
T1401
BioMarin Pharmaceutical Inc
2006 - EUCTR2005-003778-13-DE Germany;
BioMarin Pharmaceutical Inc.
2006 Phase 3 EUCTR2006-000839-10-GB Germany;Ireland;Italy;Spain;United Kingdom;
2006 Phase 3 EUCTR2005-003777-24-GB Germany;Spain;United Kingdom;
2006 - EUCTR2006-000839-10-IE 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 - EUCTR2005-003777-24-ES Germany;Spain;
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 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 - EUCTR2004-004513-41-DE Germany;Ireland;Italy;United Kingdom;
2005 - EUCTR2004-002071-16-DE Germany;Italy;United Kingdom;
- Phase 2 EUCTR2004-002071-16-IE Germany;Ireland;Italy;United Kingdom;
- - EUCTR2004-004512-23-DE 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 - EUCTR2009-015844-41-ES Germany;Spain;United Kingdom;
Merck Serono S.A. - Geneva
2011 Phase 3 EUCTR2009-015768-33-NL Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
2011 - EUCTR2009-015768-33-PT Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
ORPHANETICS Pharma Entwicklungs- GmbH
2006 - EUCTR2006-000648-15-AT Austria;
University of Massachusetts, Amherst
2019 Phase 1 NCT04014712 United States;
Tetrahydrobiopterin (BH4)
Orphanetics Pharma Entwicklungs GmbH
2007 Phase 2/Phase 3 NCT00432822 -
The University of Texas Medical Branch, Galveston
2005 Phase 1 NCT00244218 United States;
PTC Therapeutics, Inc.
2022 Phase 3 EUCTR2021-000474-29-PT 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;
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;
Taipei Veterans General Hospital, Taiwan
2020 Phase 4 NCT04227080 -
BMN 165
BioMarin Pharmaceutical
2013 Phase 3 NCT01819727 United States;
BioMarin Pharmaceutical Inc./CMIC Co., Ltd.
2019 Phase 3 JPRN-JapicCTI-194642 Japan;
BMN 165 (rAvPAL-PEG)
BioMarin Pharmaceutical
2012 Phase 2 NCT01560286 United States;
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;
Blood samples
University Hospital, Tours
2021 - NCT04879277 France;
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
Children's Research Institute
2011 - NCT01412437 United States;
Dietary Supplement for PKU patients
University Hospital, Tours
2019 - NCT03924180 France;
Free amino acids intake
University of British Columbia
2019 - NCT03939052 Canada;
Glycomacropeptide-based protein substitute
Nutricia UK Ltd
2016 - NCT02915510 United Kingdom;
HMI-103
Homology Medicines, Inc
2022 Phase 1 NCT05222178 United States;
Hepatocyte Transplant
Ira Fox
2022 Phase 1/Phase 2 NCT01465100 United States;
High oleic sunflower oil
Ludwig-Maximilians - University of Munich
2009 - NCT00909012 Germany;Italy;Spain;United Kingdom;
Immunosuppression
Ira Fox
2022 Phase 1/Phase 2 NCT01465100 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
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 KGaA
2013 Phase 4 EUCTR2009-015844-41-IT Germany;Italy;Spain;United Kingdom;
2013 - EUCTR2009-015844-41-ES Germany;Spain;United Kingdom;
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-NL Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
2011 - EUCTR2009-015768-33-PT Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
University Medical Center Groningen
2014 Phase 4 EUCTR2010-021343-41-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;
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;
Kuvan®
BioMarin Pharmaceutical
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;
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;
2012 Phase 3 NCT01732471 Germany;
2010 Phase 4 NCT01082328 Norway;
Kuvan® (sapropterin dihydrochloride)
Merck Serono, an affiliate of E. Merck AB
- Phase 4 EUCTR2009-012978-12-NO Norway;
University Medical Center Groningen
- Phase 4 EUCTR2021-003777-63-NL Netherlands;
Large Neutral Amino Acid Therapy
University of Southern California
2012 Phase 4 NCT01617070 United States;
Liver Biopsy
Ira Fox
2022 Phase 1/Phase 2 NCT01465100 United States;
Low calorie protein substitute
Nutricia UK Ltd
2020 - NCT04272736 -
Medikinet
Amsterdam UMC
2021 Phase 4 EUCTR2021-001174-29-NL Netherlands;
Microalgal oil
Ludwig-Maximilians - University of Munich
2009 - NCT00909012 Germany;Italy;Spain;United Kingdom;
Moxifloxacin
BioMarin Pharmaceutical
2008 Phase 1 NCT00789568 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-NL Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
2011 - EUCTR2009-015768-33-PT Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
PKU Explore
Vitaflo International, Ltd
2017 - NCT03168399 United Kingdom;
PKU Sphere
Vitaflo International, Ltd
2018 - NCT03771391 Germany;
2018 - NCT03419819 United States;
PKU Start
Vitaflo International, Ltd
2017 - NCT03058848 United Kingdom;
PKU explore
Vitaflo International, Ltd
2021 - NCT04898829 France;
PKU express plus
Vitaflo International, Ltd
2021 - NCT05051657 United Kingdom;
PKU sphere
Vitaflo International, Ltd
2020 - NCT04679467 Italy;
2019 - NCT04224142 United Kingdom;
PKU sphere liquid
Vitaflo International, Ltd
2021 - NCT05096988 United Kingdom;
PKU/TYR GMP Protein Substitute
Nutricia UK Ltd
2020 - NCT05062226 United Kingdom;
PTC923
PTC Therapeutics
2022 Phase 3 NCT05166161 Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;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.
2022 Phase 3 EUCTR2021-000474-29-PT 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;
Palynziq
Emory University
2019 - NCT04404530 United States;
Pegvaliase
BioMarin Pharmaceutical
2018 Phase 3 NCT03694353 United States;
BioMarin Pharmaceutical Inc./CMIC Co., Ltd.
2019 Phase 3 JPRN-JapicCTI-194642 Japan;
Boston Children's Hospital
2019 - NCT03792451 United States;
Phe
BioMarin Pharmaceutical
2011 Phase 3 NCT01376908 Austria;Belgium;Czech Republic;Czechia;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
Ira Fox
2022 Phase 1/Phase 2 NCT01465100 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 Phase 3 EUCTR2006-000839-10-GB Germany;Ireland;Italy;Spain;United Kingdom;
2006 Phase 3 EUCTR2005-003777-24-GB Germany;Spain;United Kingdom;
2006 - EUCTR2006-000839-10-IE 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 - EUCTR2005-003777-24-ES Germany;Spain;
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 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 - EUCTR2004-004513-41-DE Germany;Ireland;Italy;United Kingdom;
2005 - EUCTR2004-002071-16-DE Germany;Italy;United Kingdom;
- Phase 2 EUCTR2004-002071-16-IE Germany;Ireland;Italy;United Kingdom;
- - EUCTR2004-004512-23-DE Germany;Ireland;Italy;United Kingdom;
Phenylalanine
BioMarin Pharmaceutical
2011 Phase 3 NCT01376908 Austria;Belgium;Czech Republic;Czechia;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
1984 - NCT00065299 United States;
National Center for Research Resources (NCRR)
1983 - NCT00006142 -
University Hospital Inselspital, Berne
2019 Phase 4 NCT03788343 Switzerland;
Phenylalanine restricted diet
Assiut University
2020 - NCT04302194 -
Phenylalanine-free protein substitute in tablet form (XPhe minis)
metaX Institut fuer Diatetik GmbH
2021 - NCT04375592 United Kingdom;
Phenylalanine-free protein substitute tablets
Nutricia UK Ltd
2019 - NCT03333720 -
Phenylketonuria-type diet
University of Glasgow
2014 - NCT02440932 United Kingdom;
Protein intake
University of British Columbia
2013 - NCT01965691 Canada;
RAvPAL-PEG
BioMarin Pharmaceutical
2011 Phase 2 NCT01212744 United States;
2009 Phase 2 NCT00925054 United States;
2009 Phase 2 NCT00924703 United States;
2008 Phase 1 NCT00634660 United States;
RTX-134
Rubius Therapeutics
2020 Phase 1 NCT04110496 United States;
SAPROPTERIN DIHYDROCHLORIDE
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;
SAPROPTERIN HYDROCHLORIDE
Merck Serono Norway
2010 - EUCTR2009-018168-81-DK Denmark;
- Phase 4 EUCTR2009-018168-81-NO Denmark;Norway;
SYNB1618
Synlogic
2020 Phase 2 NCT04534842 United States;
2018 Phase 1/Phase 2 NCT03516487 United States;
SYNB1934
Synlogic
2020 Phase 2 NCT04534842 United States;
Sapropterin
BIOMARIN
2006 - EUCTR2006-000839-10-IT Germany;Ireland;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;
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;
Sapropterin (Kuvan)
Washington University School of Medicine
2008 - NCT00730080 United States;
Sapropterin Dihydrochloride
Merck Serono S.A. - Geneva
2011 Phase 3 EUCTR2009-015768-33-NL Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
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 - EUCTR2009-015768-33-PT Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
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 Phase 3 EUCTR2006-000839-10-GB Germany;Ireland;Italy;Spain;United Kingdom;
2006 Phase 3 EUCTR2005-003777-24-GB Germany;Spain;United Kingdom;
2006 - EUCTR2006-000839-10-IE 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 - EUCTR2005-003777-24-ES Germany;Spain;
2006 - EUCTR2005-003777-24-DE Germany;Spain;
Children's Research Institute
2011 - NCT01412437 United States;
Tulane University School of Medicine
2011 - NCT01274026 United States;
Sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
BioMarin Pharmaceutical
2005 Phase 3 NCT00104247 United States;
Sapropterin hydrochloride
BIOMARIN
2005 - EUCTR2004-002071-16-IT Germany;Italy;United Kingdom;
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;
- Phase 2 EUCTR2004-002071-16-IE Germany;Ireland;Italy;United Kingdom;
- - EUCTR2004-004512-23-DE 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;
Synergy
Nutricia UK Ltd
2017 - NCT03167697 United Kingdom;
T1401
BioMarin Pharmaceutical Inc
2006 - EUCTR2005-003778-13-DE Germany;
BioMarin Pharmaceutical Inc.
2006 Phase 3 EUCTR2006-000839-10-GB Germany;Ireland;Italy;Spain;United Kingdom;
2006 Phase 3 EUCTR2005-003777-24-GB Germany;Spain;United Kingdom;
2006 - EUCTR2006-000839-10-IE 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 - EUCTR2005-003777-24-ES Germany;Spain;
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 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 - EUCTR2004-004513-41-DE Germany;Ireland;Italy;United Kingdom;
2005 - EUCTR2004-002071-16-DE Germany;Italy;United Kingdom;
- Phase 2 EUCTR2004-002071-16-IE Germany;Ireland;Italy;United Kingdom;
- - EUCTR2004-004512-23-DE 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 - EUCTR2009-015844-41-ES Germany;Spain;United Kingdom;
Merck Serono S.A. - Geneva
2011 Phase 3 EUCTR2009-015768-33-NL Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
2011 - EUCTR2009-015768-33-PT Austria;Belgium;Czech Republic;Germany;Italy;Netherlands;Portugal;Slovakia;Turkey;United Kingdom;
ORPHANETICS Pharma Entwicklungs- GmbH
2006 - EUCTR2006-000648-15-AT Austria;
University of Massachusetts, Amherst
2019 Phase 1 NCT04014712 United States;
Tetrahydrobiopterin (BH4)
Orphanetics Pharma Entwicklungs GmbH
2007 Phase 2/Phase 3 NCT00432822 -
The University of Texas Medical Branch, Galveston
2005 Phase 1 NCT00244218 United States;