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ClinicalTrials.gov public records Last synced Oct 30, 2026, 1:37 PM EDT

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Showing 1–24 of 96 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: PKU Clear all
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 18, 2018 · Synced Oct 30, 2026, 1:37 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks

NCT01560286
View directory record Official record
Conditions
Phenylketonuria
Interventions
BMN 165 (rAvPAL-PEG)
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 70 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
6
States / cities
Aurora, Colorado • Gainesville, Florida • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2019 · Synced Oct 30, 2026, 1:37 PM EDT
Completed No phase listed Observational

Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU

NCT05356377
View directory record Official record
Conditions
Phenylketonurias
Interventions
Pegvaliase-Pqpz
Drug
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Boston, Massachusetts • Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Dec 29, 2025 · Synced Oct 30, 2026, 1:37 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria

NCT03097250
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
12 Years to 25 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 19, 2018 · Synced Oct 30, 2026, 1:37 PM 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 Oct 28, 2026 · Synced Oct 30, 2026, 1:37 PM EDT
Recruiting Phase 3 Interventional

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

NCT06628128
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
JNT-517
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
4 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
8
States / cities
Gainesville, Florida • Tampa, Florida • Portland, Oregon + 4 more
Source: ClinicalTrials.gov public record
Updated May 1, 2026 · Synced Oct 30, 2026, 1:37 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 5, 2013 · Synced Oct 30, 2026, 1:37 PM 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 30, 2026, 1:37 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 30, 2026, 1:37 PM 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 30, 2026, 1:37 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 30, 2026, 1:37 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 9, 2007 · Synced Oct 30, 2026, 1:37 PM EDT
Terminated Phase 1 Interventional

Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

NCT04110496
View directory record Official record
Conditions
Phenylketonurias
Interventions
RTX-134
Drug
Lead sponsor
Rubius Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Dec 8, 2022 · Synced Oct 30, 2026, 1:37 PM EDT
Recruiting Phase 3 Interventional

A Study of JNT-517 in Participants With Phenylketonuria (PKU)

NCT06971731
View directory record Official record
Conditions
Phenylketonuria
Interventions
JNT-517, JNT-517 Matching Placebo
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
4 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
Source: ClinicalTrials.gov public record
Updated Oct 21, 2026 · Synced Oct 30, 2026, 1:37 PM EDT
Completed No phase listed Observational

A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults

NCT03505125
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
4
States / cities
Gainesville, Florida • Decatur, Georgia • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 14, 2020 · Synced Oct 30, 2026, 1:37 PM EDT
Recruiting No phase listed Observational

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

NCT06305234
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2033
U.S. locations
11
States / cities
Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 30, 2026, 1:37 PM EDT
Recruiting Phase 2 Interventional

A Study of MZE782 in Adults With PKU

NCT07694440
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
MZE782, Placebo
Drug · Other
Lead sponsor
Maze Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
7
States / cities
Oakland, California • Palo Alto, California • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2026 · Synced Oct 30, 2026, 1:37 PM 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 30, 2026, 1:37 PM EDT
Terminated Phase 1Phase 2 Interventional

Liver Cell Transplant for Phenylketonuria

NCT01465100
View directory record Official record
Conditions
Phenylketonuria
Interventions
Preparative Radiation Therapy, Hepatocyte Transplant, Immunosuppression, Liver Evaluation, Neuro-psychological Assessment, Whole body Phe oxidation testing, Liver Biopsy
Radiation · Procedure · Drug + 3 more
Lead sponsor
Ira Fox
Other
Eligibility
14 Years to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 16, 2023 · Synced Oct 30, 2026, 1:37 PM 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 30, 2026, 1:37 PM EDT
Completed No phase listed Observational

Stress, Hypothalamic-pituitary-adrenal (HPA) Dysfunction, and Relapse in Alcoholism

NCT00744588
View directory record Official record
Conditions
Alcohol Dependence
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 60 Years · Male only
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 3, 2013 · Synced Oct 30, 2026, 1:37 PM EDT
Completed No phase listed Observational

Bone Mineral Density in Adults With Hyperphenylalaninemia

NCT01209819
View directory record Official record
Conditions
Phenylketonurias
Interventions
Not listed
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 12, 2015 · Synced Oct 30, 2026, 1:37 PM EDT
Completed No phase listed Observational

PheCheck Feasibility Study

NCT05998109
View directory record Official record
Conditions
Phenylketonurias
Interventions
PheCheck™, HPLC Amino Acid Analyzer, Dried Blood Spot Cards
Device · Diagnostic Test
Lead sponsor
Lumos Diagnostics
Industry
Eligibility
10 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 30, 2026, 1:37 PM EDT
Completed Not applicable Interventional

Simplified Diet Approach in Phenylketonuria

NCT02555579
View directory record Official record
Conditions
Phenylketonuria
Interventions
Dietary Protein Counting and Free Fruits and Vegetables
Behavioral
Lead sponsor
University of Nebraska
Other
Eligibility
13 Years to 65 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Omaha, Nebraska
Source: ClinicalTrials.gov public record
Updated Sep 7, 2023 · Synced Oct 30, 2026, 1:37 PM EDT