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ClinicalTrials.gov public records Last synced Oct 7, 2026, 8:02 AM EDT

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Showing 49–72 of 95 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Phenylketonuria (PKU) Clear all
Completed Phase 4 Interventional Results available

The Effectiveness of Kuvan in Amish PKU Patients

NCT02677870
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 27, 2022 · Synced Oct 7, 2026, 8:02 AM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria

NCT03516487
View directory record Official record
Conditions
Phenylketonuria, Healthy
Interventions
SYNB1618, Placebo
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years to 64 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
4
States / cities
Boston, Massachusetts • Pittsburgh, Pennsylvania • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated May 13, 2021 · Synced Oct 7, 2026, 8:02 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 7, 2026, 8:02 AM 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 7, 2026, 8:02 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 7, 2026, 8:02 AM 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
6
States / cities
Oakland, California • Palo Alto, California • Indianapolis, Indiana + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Oct 7, 2026, 8:02 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 7, 2026, 8:02 AM EDT
Terminated Phase 2 Interventional Accepts healthy volunteers Results available

Phenylketonuria, Oxidative Stress, and BH4

NCT01395394
View directory record Official record
Conditions
Phenylketonuria
Interventions
Kuvan, Meal Challenge
Drug · Other
Lead sponsor
Emory University
Other
Eligibility
10 Years to 45 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Aug 28, 2014 · Synced Oct 7, 2026, 8:02 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 7, 2026, 8:02 AM EDT
Terminated Phase 1Phase 2 Interventional

Gene Therapy Clinical Study in Adult PKU

NCT03952156
View directory record Official record
Conditions
Phenylketonurias, PAH Deficiency
Interventions
HMI-102
Genetic
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
13
States / cities
Los Angeles, California • Orange, California • Tampa, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2023 · Synced Oct 7, 2026, 8:02 AM 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 7, 2026, 8:02 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 7, 2026, 8:02 AM EDT
Recruiting Phase 1Phase 2 Interventional

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

NCT06332807
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 7, 2026, 8:02 AM 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 7, 2026, 8:02 AM EDT
Completed Not applicable Interventional

Evaluation of PKU Sphere

NCT03419819
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 16, 2024 · Synced Oct 7, 2026, 8:02 AM EDT
Completed Not applicable Interventional Results available

Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria

NCT01428258
View directory record Official record
Conditions
Phenylketonuria
Interventions
GMP Diet/GMP Medical Foods, AA Diet/AA Medical Foods
Other
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
12 Years to 45 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
2
States / cities
Boston, Massachusetts • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Aug 24, 2018 · Synced Oct 7, 2026, 8:02 AM EDT
Recruiting No phase listed Observational

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

NCT05813678
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2033
U.S. locations
12
States / cities
Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 7, 2026, 8:02 AM 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 7, 2026, 8:02 AM 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 7, 2026, 8:02 AM EDT
Recruiting No phase listed Observational

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

NCT05579548
View directory record Official record
Conditions
Phenylketonuria, Maternal
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Female only
Enrollment
50 participants
Timeline
2022 – 2032
U.S. locations
1
States / cities
Wilmington, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 7, 2026, 8:02 AM EDT
Recruiting Phase 4 Interventional

Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

NCT07477691
View directory record Official record
Conditions
Phenylketonuria
Interventions
Pegvaliase, Methotrexate
Biological · Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
12
States / cities
Los Angeles, California • Aurora, Colorado • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Oct 7, 2026, 8:02 AM 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 7, 2026, 8:02 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 7, 2026, 8:02 AM EDT
Recruiting Not applicable Interventional

Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

NCT06560736
View directory record Official record
Conditions
Phenylketonurias
Interventions
Show Me FIRST coping skills intervention
Behavioral
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
10 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 7, 2026, 8:02 AM EDT