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ClinicalTrials.gov public records Last synced Oct 9, 2026, 11:40 PM EDT

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Showing 73–95 of 95 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Phenylketonuria (PKU) Clear all
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 9, 2026, 11:40 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 12, 2024 · Synced Oct 9, 2026, 11:40 PM 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 9, 2026, 11:40 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 11, 2015 · Synced Oct 9, 2026, 11:40 PM 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 9, 2026, 11:40 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 9, 2026, 11:40 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

NCT05781399
View directory record Official record
Conditions
Phenylketonuria
Interventions
JNT-517 Suspension, Placebo Suspension, JNT-517 Tablet, Placebo Tablet
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
163 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
11
States / cities
Gainesville, Florida • Tampa, Florida • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 9, 2026, 11:40 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 20, 2016 · Synced Oct 9, 2026, 11:40 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 9, 2026, 11:40 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 9, 2026, 11:40 PM EDT
Completed Phase 3 Interventional Results available

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

NCT01819727
View directory record Official record
Conditions
Phenylketonuria
Interventions
BMN 165
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
261 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
31
States / cities
La Jolla, California • Oakland, California • Aurora, Colorado + 28 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 9, 2026, 11:40 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 9, 2026, 11:40 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 25, 2020 · Synced Oct 9, 2026, 11:40 PM 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 9, 2026, 11:40 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 10, 2023 · Synced Oct 9, 2026, 11:40 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 28, 2021 · Synced Oct 9, 2026, 11:40 PM EDT
Completed Not applicable Interventional

The Effect of Supplemental Docosahexaenoic Acid (DHA) on Neurocognitive Outcomes in Teen and Adult Women With Phenylketonuria(PKU)

NCT00892554
View directory record Official record
Conditions
Phenylketonuria
Interventions
Docosahexaenoic Acid, Corn/soy oil
Dietary Supplement
Lead sponsor
Emory University
Other
Eligibility
12 Years to 50 Years · Female only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 11, 2015 · Synced Oct 9, 2026, 11:40 PM EDT
Not listed Not applicable Interventional

Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU)

NCT05174559
View directory record Official record
Conditions
Phenylketonurias
Interventions
PreKUnil® LNAA Medical Food for PKU
Other
Lead sponsor
University of Southern California
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 4, 2023 · Synced Oct 9, 2026, 11:40 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 9, 2026, 11:40 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 9, 2026, 11:40 PM 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 9, 2026, 11:40 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 18, 2013 · Synced Oct 9, 2026, 11:40 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 9, 2026, 11:40 PM EDT