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Showing 1–24 of 61 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: Sapropterin (Kuvan) Clear all
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Long Term Nitric Oxide Bioavailability on Vascular Health in Chronic Obstructive Pulmonary Disease

NCT02774226
View directory record Official record
Conditions
Chronic Obstructive Pulmonary Disease (COPD)
Interventions
Tetrahydrobiopterin (BH4), Antioxidant Cocktail
Drug · Dietary Supplement
Lead sponsor
Augusta University
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2018
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 22, 2021 · Synced Sep 21, 2026, 5:35 PM EDT
Terminated No phase listed Observational

Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

NCT00827762
View directory record Official record
Conditions
Phenylketonuria
Interventions
Kuvan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
6 Years to 18 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
4
States / cities
Chicago, Illinois • Columbia, Missouri • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2013 · Synced Sep 21, 2026, 5:35 PM EDT
Completed No phase listed Observational

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

NCT00778206
View directory record Official record
Conditions
Phenylketonuria, Hyperphenylalaninaemia
Interventions
Kuvan
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
1,887 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2021
U.S. locations
45
States / cities
Los Angeles, California • Orange, California • San Diego, California + 38 more
Source: ClinicalTrials.gov public record
Updated Oct 11, 2022 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 3 Interventional Results available

Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

NCT01114737
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin dihydrochloride, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years to 65 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
26
States / cities
La Jolla, California • Los Angeles, California • Palo Alto, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2016 · Synced Sep 21, 2026, 5:35 PM EDT
Terminated Phase 2 Interventional Results available

Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction

NCT00802893
View directory record Official record
Conditions
Isolated Systolic Hypertension, Endothelial Dysfunction
Interventions
6R-BH4, Placebo
Drug · Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
40 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 20, 2017 · Synced Sep 21, 2026, 5:35 PM EDT
Recruiting Phase 2 Interventional

Neurovascular Transduction During Exercise in Chronic Kidney Disease

NCT02947750
View directory record Official record
Conditions
Renal Insufficiency, Chronic
Interventions
6R-BH4, 6R-BH4 placebo, Folic acid, Exercise training, Stretching, Histidine and beta-alanine supplementation, Histidine and beta-alanine placebo
Drug · Other · Dietary Supplement
Lead sponsor
Emory University
Other
Eligibility
18 Years to 75 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2027
U.S. locations
3
States / cities
Atlanta, Georgia • Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated May 17, 2026 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Systemic Sclerosis (SSc) Vasculopathy: Improved Clinical Monitoring and Treatment

NCT02530996
View directory record Official record
Conditions
Rheumatologic Disease
Interventions
BH4, Vasculopathy assessment, Placebo
Drug · Diagnostic Test
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2019
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Apr 4, 2021 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

NCT03519711
View directory record Official record
Conditions
BH4 Deficiency, Hyperphenylalaninemia
Interventions
PTC923
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
12 Months and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
4
States / cities
Minneapolis, Minnesota • Dallas, Texas • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 13, 2023 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Kuvan in People With Schizophrenia and Schizoaffective Disorder

NCT01706965
View directory record Official record
Conditions
Schizophrenia, Schizoaffective Disorder
Interventions
Kuvan, Multivitamin
Drug
Lead sponsor
New York State Psychiatric Institute
Other
Eligibility
18 Years to 64 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 12, 2017 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Cystic Fibrosis and Endothelial Function: At Rest and During Exercise

NCT01772758
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
BH4 5mg, BH4 20mg, Antioxidant Cocktail
Drug · Dietary Supplement
Lead sponsor
Augusta University
Other
Eligibility
7 Years and older
Enrollment
64 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2016
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Jun 19, 2019 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 2 Interventional Results available

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

NCT00532844
View directory record Official record
Conditions
Endothelial Dysfunction
Interventions
Sapropterin Dihydrochloride, Sapropterin Dihydrochloride and Vitamin C
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 75 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Hackensack, New Jersey
Source: ClinicalTrials.gov public record
Updated May 16, 2021 · Synced Sep 21, 2026, 5:35 PM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers

Response to Phenylketonuria to Tetrahydrobiopterin (BH4)

NCT00244218
View directory record Official record
Conditions
Phenylketonuria
Interventions
tetrahydrobiopterin (BH4)
Drug
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
10 Years and older
Enrollment
57 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2015
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 20, 2021 · Synced Sep 21, 2026, 5:35 PM EDT
Withdrawn Phase 2 Interventional

L-arginine Study for Persistent Symptoms of Schizophrenia

NCT04054973
View directory record Official record
Conditions
Schizo Affective Disorder, Schizophrenia
Interventions
L-arginine, Sapropterin Dihydrochloride
Drug
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 19, 2022 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of Kuvan Treatment in Adults With GTPCH Deficiency

NCT01425528
View directory record Official record
Conditions
GTP Cyclohydrolase Deficiency
Interventions
Sapropterin
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Mar 31, 2025 · Synced Sep 21, 2026, 5:35 PM EDT
Recruiting Phase 1 Interventional

Novel Approaches for Improving Vascular Function in Veterans With HFpEF

NCT06312748
View directory record Official record
Conditions
Heart Failure With Preserved Ejection Fraction, Inflammation
Interventions
L-Citrulline, Placebo for L-Citrulline, BH4, Placebo for BH4, Atorvastatin, Placebo for Atorvastatin
Drug
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

NCT00104247
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
13
States / cities
Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2014 · Synced Sep 21, 2026, 5:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated Phenylketonuria

NCT02297347
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 4, 2018 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Nitric Oxide Bioavailability in Chronic Obstructive Pulmonary Disease (COPD)

NCT01398943
View directory record Official record
Conditions
Pulmonary Disease, Chronic Obstructive
Interventions
Tetrahydrobiopterin (BH4), Antioxidant Cocktail
Drug · Dietary Supplement
Lead sponsor
Augusta University
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2015
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 10, 2017 · Synced Sep 21, 2026, 5:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Sapropterin in Individuals With Phenylketonuria

NCT00730080
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin (Kuvan)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
6 Years to 50 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2018
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 17, 2018 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Open-Label Extension Study of Kuvan for Autism

NCT00943579
View directory record Official record
Conditions
Autistic Disorder
Interventions
Kuvan®
Drug
Lead sponsor
The Children's Health Council
Other
Eligibility
3 Years to 6 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated May 1, 2018 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 2 Interventional

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

NCT00104260
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
13
States / cities
Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2007 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Exercise Intolerance in Renal Failure

NCT01356966
View directory record Official record
Conditions
Kidney Failure, Chronic
Interventions
Tetrahydrobiopterin, Placebo, Folate
Drug · Dietary Supplement
Lead sponsor
Emory University
Other
Eligibility
18 Years to 75 Years
Enrollment
74 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
2
States / cities
Atlanta, Georgia • Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated May 31, 2015 · Synced Sep 21, 2026, 5:35 PM EDT
Completed Phase 4 Interventional Results available

Effects of Kuvan on Melatonin Secretion

NCT01617070
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Kuvan, Large Neutral Amino Acid Therapy
Drug · Dietary Supplement
Lead sponsor
University of Southern California
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 17, 2016 · Synced Sep 21, 2026, 5:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders

NCT01274026
View directory record Official record
Conditions
Phenylketonuria, Behavior and Behavior Mechanisms, PAH Gene Expression
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
Tulane University School of Medicine
Other
Eligibility
2 Years to 21 Years
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Aug 13, 2023 · Synced Sep 21, 2026, 5:35 PM EDT