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ClinicalTrials.gov public records Last synced Oct 4, 2026, 1:32 AM EDT

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Showing 25–48 of 95 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Phenylketonurias Clear all
Completed Phase 2 Interventional

A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

NCT00325962
View directory record Official record
Conditions
Hypertension
Interventions
6R-BH4 (sapropterin dihydrochloride)
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
24
States / cities
Hoover, Alabama • Huntsville, Alabama • Mobile, Alabama + 21 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2009 · Synced Oct 4, 2026, 1:32 AM 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 Feb 1, 2016 · Synced Oct 4, 2026, 1:32 AM EDT
Completed Not applicable Interventional Results available

The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study

NCT01917344
View directory record Official record
Conditions
Phenylketonuria
Interventions
MRI
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 19, 2015 · Synced Oct 4, 2026, 1:32 AM EDT
Recruiting Phase 1 Interventional

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

NCT07241234
View directory record Official record
Conditions
Phenylketonuria
Interventions
AG-181
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 69 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Indianapolis, Indiana • Pittsburgh, Pennsylvania • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 4, 2026, 1:32 AM EDT
Completed No phase listed Observational

Nutrition Status of Adults Treated With Pegvaliase

NCT03792451
View directory record Official record
Conditions
Phenylketonurias
Interventions
Pegvaliase
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 4, 2021 · Synced Oct 4, 2026, 1:32 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects

NCT00789568
View directory record Official record
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride, Moxifloxacin, Moxifloxacin placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 50 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Fargo, North Dakota
Source: ClinicalTrials.gov public record
Updated Jul 23, 2021 · Synced Oct 4, 2026, 1:32 AM EDT
Completed Phase 3 Interventional

A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

NCT00225615
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
14
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2009 · Synced Oct 4, 2026, 1:32 AM EDT
Terminated Not applicable Interventional

Moderate Intensity Exercise and Phenylketonuria

NCT01904708
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 16, 2014 · Synced Oct 4, 2026, 1:32 AM EDT
Completed No phase listed Observational

A Prospective Clinical Study of Phenylketonuria (PKU)

NCT04452513
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 14, 2023 · Synced Oct 4, 2026, 1:32 AM EDT
Completed Phase 1 Interventional

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

NCT00634660
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
8
States / cities
Chicago, Illinois • Minneapolis, Minnesota • St Louis, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2017 · Synced Oct 4, 2026, 1:32 AM 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 4, 2026, 1:32 AM EDT
Completed No phase listed Observational Results available

A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

NCT02468570
View directory record Official record
Conditions
Phenylketonuria
Interventions
Administration of CANTAB and Subject Global Assessment
Other
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Lexington, Kentucky • Omaha, Nebraska • Oklahoma City, Oklahoma + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2020 · Synced Oct 4, 2026, 1:32 AM 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 4, 2026, 1:32 AM 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 4, 2026, 1:32 AM EDT
Completed Phase 3 Interventional Results available

A Study of PTC923 in Participants With Phenylketonuria

NCT05099640
View directory record Official record
Conditions
Phenylketonuria
Interventions
PTC923, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
12
States / cities
Stanford, California • Aurora, Colorado • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Oct 4, 2026, 1:32 AM 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 4, 2026, 1:32 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Remote Neurocognitive and Psychological Assessment in PKU

NCT04943393
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 22, 2022 · Synced Oct 4, 2026, 1:32 AM EDT
Completed No phase listed Observational

Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

NCT03856203
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Oct 4, 2026, 1:32 AM EDT
Completed Phase 2 Interventional Results available

Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response

NCT00841100
View directory record Official record
Conditions
Phenylketonuria
Interventions
Kuvan, Diet
Drug · Other
Lead sponsor
University of Miami
Other
Eligibility
4 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced Oct 4, 2026, 1:32 AM EDT
Completed Phase 3 Interventional Results available

Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

NCT00332189
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 16, 2012 · Synced Oct 4, 2026, 1:32 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

AAV Gene Therapy Study for Subjects with PKU

NCT04480567
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 12, 2024 · Synced Oct 4, 2026, 1:32 AM 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 17, 2021 · Synced Oct 4, 2026, 1:32 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers

NCT07220265
View directory record Official record
Conditions
Carrier of Phenylketonuria, Healthy
Interventions
Phenylalanine (Phe), Placebo
Dietary Supplement
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 60 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Oct 4, 2026, 1:32 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU)

NCT00065299
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 4, 2026, 1:32 AM EDT