Independent directory Public ClinicalTrials.gov records United States
Clear filters
ClinicalTrials.gov public records Last synced Jul 22, 2026, 12:09 PM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Completed Not applicable Interventional

Evaluation of PKU Sphere

NCT03419819
Conditions
Phenylketonurias
Interventions
PKU Sphere
Dietary Supplement
Lead sponsor
Vitaflo International, Ltd
Industry
Eligibility
3 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Portland, Oregon
Conditions
Phenylketonurias
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Phenylketonurias
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
14 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
14
States / cities
Aurora, Colorado • Gainesville, Florida • Tampa, Florida + 11 more
Active, not recruiting Phase 1Phase 2 Interventional

AAV Gene Therapy Study for Subjects with PKU

NCT04480567
Conditions
Phenylketonuria (PKU)
Interventions
BMN 307
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
15 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
2
States / cities
Tampa, Florida • Morristown, New Jersey
Conditions
Phenylketonuria, PKU
Interventions
Acute Moderate Intensity Exercise
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
14 Years to 17 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Portland, Oregon
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
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 6 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
12
States / cities
La Jolla, California • Orange, California • Tampa, Florida + 9 more
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
Completed Not applicable Interventional Accepts healthy volunteers

Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU)

NCT00065299
Conditions
Phenylketonuria
Interventions
Restricted phenylalanine diet
Behavioral
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
0 Years and older · Female only
Enrollment
572 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1984 – 2000
U.S. locations
3
States / cities
Los Angeles, California • Chicago, Illinois • Galveston, Texas
Conditions
Phenylketonuria
Interventions
JNT-517 Tablet, Placebo Tablet: BID
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
10
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 7 more
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
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
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 55 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2019
U.S. locations
14
States / cities
Aurora, Colorado • Gainesville, Florida • Chicago, Illinois + 11 more
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
4 Years and older
Enrollment
111 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
14
States / cities
Los Angeles, California • Sacramento, California • San Jose, California + 11 more
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Gainesville, Florida • Tampa, Florida • Chicago, Illinois + 14 more
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
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
Completed No phase listed Observational Accepts healthy volunteers

Sapropterin in Individuals With Phenylketonuria

NCT00730080
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
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase, Diet Only
Drug · Other
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
12 Years to 17 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Aurora, Colorado + 10 more
Conditions
Phenylketonurias
Interventions
NGGT002
Genetic
Lead sponsor
NGGT INC.
Industry
Eligibility
18 Years to 55 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
5
States / cities
Orange, California • Minneapolis, Minnesota • Morristown, New Jersey + 2 more
Completed No phase listed Observational Accepts healthy volunteers

Remote Neurocognitive and Psychological Assessment in PKU

NCT04943393
Conditions
Phenylketonurias
Interventions
nointervention
Other
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 40 Years
Enrollment
84 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia • Columbia, Missouri
Conditions
Phenylketonuria
Interventions
PTC923
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
7
States / cities
Aurora, Colorado • Gainesville, Florida • Indianapolis, Indiana + 4 more
Conditions
Pulmonary Arterial Hypertension (PAH)
Interventions
Allogeneic Human Cardiosphere-Derived Stem Cells, Placebo
Biological
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Pulmonary Arterial Hypertension, Familial Primary Pulmonary Hypertension, Primary Pulmonary Hypertension, Lung Diseases, Carnitine Nutritional Deficiency
Interventions
L-carnitine
Dietary Supplement
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years to 85 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Nashville, Tennessee