Independent directory Public ClinicalTrials.gov records United States
Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 8, 2026, 10:06 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 49–61 of 61 matching trials from the live ClinicalTrials.gov search.
Completed Phase 1Phase 2 Interventional Results available

Study of Kuvan Treatment in Adults With GTPCH Deficiency

NCT01425528
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
Completed Phase 4 Interventional Results available

The Effectiveness of Kuvan in Amish PKU Patients

NCT02677870
Conditions
Phenylketonuria
Interventions
saproterin dihydrochloride, Diet treatment
Drug · Other
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
2 Years to 60 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Cleveland, Ohio
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
Completed Phase 2Phase 3 Interventional Results available

Open-Label Extension Study of Kuvan for Autism

NCT00943579
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
Completed Phase 1 Interventional Accepts healthy volunteers

The Effect of Antioxidants on Skin Blood Flow-BH4

NCT03680573
Conditions
Cardiovascular Diseases, Cardiovascular Risk Factor, Vasoconstriction
Interventions
Control: Lacated Ringers, Apocynin, Allopurinol, BH4, NG Nitro L Arginine Methyl Ester, Sodium Nitroprusside, Acetyl-ß-methylcholine chloride
Drug
Lead sponsor
The University of Texas at Arlington
Other
Eligibility
18 Years to 35 Years
Enrollment
16 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Arlington, Texas
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

NCT01541397
Conditions
Hyperphenylalaninemia, Phenylketonuria
Interventions
Sapropterin
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years to 50 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
1
States / cities
Houston, Texas
Conditions
Gastroparesis
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
Stanford University
Other
Eligibility
18 Years to 65 Years · Female only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Stanford, California
Completed Early Phase 1 Interventional Accepts healthy volunteers

Sex Hormones and Atherosclerosis Prevention in Perimenopausal Women

NCT02042196
Conditions
Menopause, Aging
Interventions
KUVAN, Placebo, Cetrotide, Climara, Placebo transdermal patch
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 70 Years · Female only
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Aurora, Colorado
Conditions
Healthy
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Male only
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
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
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
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Cystic Fibrosis and Endothelial Function: At Rest and During Exercise

NCT01772758
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
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

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

NCT02530996
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