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ClinicalTrials.gov public records Last synced Sep 12, 2026, 1:29 PM EDT

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Showing 1–24 of 95 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Phenylketonuria Clear all
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 23, 2026 · Synced Sep 12, 2026, 1:29 PM 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 16, 2026 · Synced Sep 12, 2026, 1:29 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 19, 2009 · Synced Sep 12, 2026, 1:29 PM EDT
Active, not recruiting Phase 4 Interventional

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

NCT06780332
View directory record Official record
Conditions
Phenylketonuria, PKU
Interventions
RDD to Palynziq
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Chevy Chase, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced Sep 12, 2026, 1:29 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 14, 2025 · Synced Sep 12, 2026, 1:29 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 13, 2023 · Synced Sep 12, 2026, 1:29 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 2, 2013 · Synced Sep 12, 2026, 1:29 PM 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 9, 2024 · Synced Sep 12, 2026, 1:29 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 7, 2015 · Synced Sep 12, 2026, 1:29 PM 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 16, 2021 · Synced Sep 12, 2026, 1:29 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 12, 2026, 1:29 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 15, 2026 · Synced Sep 12, 2026, 1:29 PM EDT
Completed Phase 3 Interventional

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

NCT05166161
View directory record Official record
Conditions
Phenylketonuria
Interventions
PTC923
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
247 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
7
States / cities
Aurora, Colorado • Gainesville, Florida • Indianapolis, Indiana + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Sep 12, 2026, 1:29 PM EDT
Terminated Phase 3 Interventional Results available

Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3)

NCT05764239
View directory record Official record
Conditions
Phenylketonuria
Interventions
SYNB1934v1, Placebo
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
14
States / cities
Culver City, California • Orange, California • Palo Alto, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jun 19, 2024 · Synced Sep 12, 2026, 1:29 PM EDT
Recruiting No phase listed Observational

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

NCT04404530
View directory record Official record
Conditions
Phenylketonurias
Interventions
Palynziq
Drug
Lead sponsor
Emory University
Other
Eligibility
16 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Sep 12, 2026, 1:29 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 12, 2026, 1:29 PM EDT
Completed Phase 3 Interventional Results available

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

NCT01889862
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
BMN165 20mg/day, BMN165 40mg/day, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
215 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
29
States / cities
La Jolla, California • Oakland, California • Aurora, Colorado + 26 more
Source: ClinicalTrials.gov public record
Updated May 20, 2021 · Synced Sep 12, 2026, 1:29 PM 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 22, 2025 · Synced Sep 12, 2026, 1:29 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 25, 2019 · Synced Sep 12, 2026, 1:29 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 12, 2026, 1:29 PM EDT
Terminated No phase listed Observational

Natural History Clinical Study in Adult PKU

NCT04768348
View directory record Official record
Conditions
Phenylketonurias, PKU
Interventions
Not listed
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
8
States / cities
Los Angeles, California • Orange, California • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2023 · Synced Sep 12, 2026, 1:29 PM EDT
Completed Not applicable Interventional Results available

Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy

NCT00986973
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 50 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 7, 2015 · Synced Sep 12, 2026, 1:29 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

NCT00104247
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
13
States / cities
Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2014 · Synced Sep 12, 2026, 1:29 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 13, 2023 · Synced Sep 12, 2026, 1:29 PM EDT