sapropterin dihydrochloride
Sponsors
BioMarin Pharmaceutical, Stanford University, The University of Texas Health Science Center, Houston, Tulane University School of Medicine, Children's National Research Institute
Conditions
Autism Spectrum DisorderBehavior and Behavior MechanismsEndothelial DysfunctionGastroparesisIntermittent ClaudicationPAH Gene ExpressionPKUPancreatic Adenocarcinoma Metastatic
Phase 1
Phase 2
Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
CompletedNCT00104260
Start: 2004-12-31End: 2005-11-30Target: 700Updated: 2007-04-09
A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
CompletedNCT00403494
Start: 2006-12-31End: 2009-01-31Updated: 2021-01-07
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
CompletedNCT00445978
Start: 2007-05-31End: 2009-06-30Updated: 2021-02-25
A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
CompletedNCT00532844
Start: 2007-09-30End: 2009-03-31Updated: 2021-05-17
L-arginine Study for Persistent Symptoms of Schizophrenia
WithdrawnNCT04054973
Start: 2019-09-11End: 2021-12-31Updated: 2022-01-20
PD-1 Antibody and Sapropterin Dihydrochloride in Patients With PDAC
RecruitingNCT06396637
Start: 2024-04-30End: 2025-04-30Target: 20Updated: 2024-05-02
Phase 3
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
CompletedNCT00225615
Start: 2005-11-30End: 2006-06-30Target: 100Updated: 2009-08-20
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
CompletedNCT00272792
Start: 2006-02-28End: 2006-11-30Updated: 2015-08-13
Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
CompletedNCT00332189
Start: 2006-07-31End: 2009-08-31Updated: 2012-11-16
Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
CompletedNCT00838435
Start: 2009-02-28End: 2018-09-14Updated: 2020-12-22
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
CompletedNCT01114737
Start: 2010-08-31End: 2013-03-31Updated: 2016-02-01
Unknown Phase
Sapropterin Expanded Access Program
NCT00484991
Updated: 2008-04-15
Impact of KUVAN® on Gastric Relaxation in Women With Diabetic Gastroparesis.
CompletedNCT01135186
Start: 2010-05-31End: 2012-11-30Updated: 2019-09-10
Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
CompletedNCT01141595
Start: 2010-07-31End: 2011-09-30Updated: 2014-04-01
Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders
CompletedNCT01274026
Start: 2011-01-31End: 2013-12-31Updated: 2023-08-14
Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria
WithdrawnNCT01412437
Start: 2011-04-30End: 2012-12-31Updated: 2014-12-17
Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU
RecruitingNCT07728032
Start: 2025-10-01End: 2026-12-01Target: 148Updated: 2026-07-27