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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Phenylketonuria Clear all
Recruiting Not applicable Interventional Accepts healthy volunteers

Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria

NCT01659749
View directory record Official record
Conditions
Pregnancy, Phenylketonuria
Interventions
Metabolic camp
Behavioral
Lead sponsor
Emory University
Other
Eligibility
11 Years and older · Female only
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2037
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Sep 26, 2026, 3:20 PM EDT
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 26, 2022 · Synced Sep 26, 2026, 3:20 PM 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Withdrawn Phase 1 Interventional Accepts healthy volunteers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 18, 2023 · Synced Sep 26, 2026, 3:20 PM 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 10, 2026 · Synced Sep 26, 2026, 3:20 PM 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 18, 2015 · Synced Sep 26, 2026, 3:20 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT05972629
View directory record Official record
Conditions
Phenylketonuria
Interventions
SAR444836
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Gainesville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Sep 26, 2026, 3:20 PM EDT
Completed Phase 2 Interventional Results available

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

NCT00924703
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 11, 2021 · Synced Sep 26, 2026, 3:20 PM 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 4, 2018 · Synced Sep 26, 2026, 3:20 PM 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 3, 2021 · Synced Sep 26, 2026, 3:20 PM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

NCT05270837
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Sep 26, 2026, 3:20 PM EDT
Enrolling by invitation No phase listed Observational

Long-Term Follow Up Study of Subjects Previously Administered HMI 102

NCT04348708
View directory record Official record
Conditions
Phenylketonuria, PAH Deficiency
Interventions
HMI-102
Genetic
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 29, 2022 · Synced Sep 26, 2026, 3:20 PM 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 22, 2021 · Synced Sep 26, 2026, 3:20 PM 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 24, 2020 · Synced Sep 26, 2026, 3:20 PM EDT
Completed Phase 2 Interventional Results available

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

NCT00925054
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg, rAvPAL-PEG 0.03 mg/kg, rAvPAL-PEG 0.1 mg/kg
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
11
States / cities
Aurora, Colorado • Decatur, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2019 · Synced Sep 26, 2026, 3:20 PM EDT
Completed No phase listed Observational

Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors

NCT00728676
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Shoji Yano
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced Sep 26, 2026, 3:20 PM 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 1, 2026 · Synced Sep 26, 2026, 3:20 PM EDT
Terminated Phase 1 Interventional

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

NCT05222178
View directory record Official record
Conditions
Phenylketonurias, PAH Deficiency, Phenylketonuria
Interventions
HMI-103
Drug
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
2
States / cities
Topeka, Indiana • Lancaster, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 9, 2023 · Synced Sep 26, 2026, 3:20 PM 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 27, 2021 · Synced Sep 26, 2026, 3:20 PM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

A Pilot Study on Diurnal Variation

NCT01806051
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Kuvan
Drug
Lead sponsor
Dr. Linda Randolph
Other
Eligibility
4 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 19, 2016 · Synced Sep 26, 2026, 3:20 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 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
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Sep 26, 2026, 3:20 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

NCT03694353
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 16, 2022 · Synced Sep 26, 2026, 3:20 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Testing of Four Home Phenylalanine Monitoring Prototype Devices

NCT02445521
View directory record Official record
Conditions
Phenylketonurias
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
Not listed
Enrollment
9 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015
U.S. locations
3
States / cities
Atlanta, Georgia • Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 10, 2015 · Synced Sep 26, 2026, 3:20 PM 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 11, 2024 · Synced Sep 26, 2026, 3:20 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 28, 2025 · Synced Sep 26, 2026, 3:20 PM EDT