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ClinicalTrials.gov public records Last synced Oct 21, 2026, 3:09 PM EDT

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

Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria

NCT00625820
View directory record Official record
Conditions
Kidney Disease, Albuminuria
Interventions
6R BH4, Vitamin C
Drug · Dietary Supplement
Lead sponsor
University of Michigan
Other
Eligibility
18 Years to 75 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Oct 18, 2016 · Synced Oct 21, 2026, 3:09 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

PKU Skin Stripping

NCT04368624
View directory record Official record
Conditions
Phenylketonurias
Interventions
Skin Tape, Blood Draw
Other · Diagnostic Test
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
86 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2021
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced Oct 21, 2026, 3:09 PM EDT
Approved For Marketing No phase listed Expanded access

Sapropterin Expanded Access Program

NCT00484991
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
9 Years and older
U.S. locations
36
States / cities
Los Angeles, California • Orange, California • San Diego, California + 33 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2008 · Synced Oct 21, 2026, 3:09 PM EDT
Completed Phase 2 Interventional Results available

A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease

NCT00445978
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Sapropterin Dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
15 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
12
States / cities
Washington D.C., District of Columbia • Augusta, Georgia • Savannah, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2021 · Synced Oct 21, 2026, 3:09 PM EDT
Withdrawn Phase 2Phase 3 Interventional

Trial of Kuvan in Lesch-Nyhan Disease

NCT00935753
View directory record Official record
Conditions
Behavioral Manifestations of Lesch-Nyhan Disease
Interventions
sapropterin
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
4 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2019 · Synced Oct 21, 2026, 3:09 PM EDT
Withdrawn Not applicable Interventional

Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria

NCT01412437
View directory record Official record
Conditions
PKU
Interventions
diet, sapropterin dihydrochloride
Dietary Supplement · Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
18 Years to 40 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Dec 17, 2014 · Synced Oct 21, 2026, 3:09 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

NCT00964236
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin
Drug
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
6 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 4, 2018 · Synced Oct 21, 2026, 3:09 PM EDT
Withdrawn Phase 1 Interventional Accepts healthy volunteers

O2 Transport and Utilization in Health and Lung Disease

NCT04014712
View directory record Official record
Conditions
COPD, Tetrahydrobiopterin Deficiency, Oxidative Stress
Interventions
Tetrahydrobiopterin, Placebo oral tablet
Drug
Lead sponsor
University of Massachusetts, Amherst
Other
Eligibility
18 Years to 85 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Amherst, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 19, 2023 · Synced Oct 21, 2026, 3:09 PM EDT
Completed Phase 2 Interventional Results available

Sapropterin as a Treatment for Autistic Disorder

NCT00850070
View directory record Official record
Conditions
Autistic Disorder
Interventions
sapropterin, Placebo
Drug
Lead sponsor
The Children's Health Council
Other
Eligibility
3 Years to 6 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated May 1, 2018 · Synced Oct 21, 2026, 3:09 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 Apr 1, 2025 · Synced Oct 21, 2026, 3:09 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 5, 2018 · Synced Oct 21, 2026, 3:09 PM EDT
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 23, 2021 · Synced Oct 21, 2026, 3:09 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 23, 2026 · Synced Oct 21, 2026, 3:09 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Nutritional and Neurotransmitter Changes in PKU Subjects on BH4

NCT00688844
View directory record Official record
Conditions
Phenylketonuria
Interventions
KuvanTM Therapy
Drug
Lead sponsor
Emory University
Other
Eligibility
4 Years and older
Enrollment
58 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2010
U.S. locations
2
States / cities
Atlanta, Georgia • Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Jun 8, 2015 · Synced Oct 21, 2026, 3:09 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 14, 2023 · Synced Oct 21, 2026, 3:09 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency

NCT00355264
View directory record Official record
Conditions
Tetrahydrobiopterin Deficiencies, Hyperphenylalaninemia, Non-Phenylketonuric
Interventions
Phenoptin
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
9
States / cities
Los Angeles, California • Chicago, Illinois • Minneapolis, Minnesota + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2020 · Synced Oct 21, 2026, 3:09 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 12, 2022 · Synced Oct 21, 2026, 3:09 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 20, 2022 · Synced Oct 21, 2026, 3:09 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 14, 2023 · Synced Oct 21, 2026, 3:09 PM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

A Pilot Study on Diurnal Variation

NCT01806051
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Kuvan
Drug
Lead sponsor
Dr. Linda Randolph
Other
Eligibility
4 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 20, 2016 · Synced Oct 21, 2026, 3:09 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 21, 2017 · Synced Oct 21, 2026, 3:09 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 11, 2017 · Synced Oct 21, 2026, 3:09 PM EDT
Completed No phase listed Observational

Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors

NCT00728676
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Shoji Yano
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 18, 2013 · Synced Oct 21, 2026, 3:09 PM EDT
Completed Not applicable Interventional Results available

Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy

NCT00986973
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 50 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 8, 2015 · Synced Oct 21, 2026, 3:09 PM EDT