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Showing 1–24 of 93 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Phenylketonuria Clear all
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 24, 2026 · Synced Jul 21, 2026, 6:07 PM EDT
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 Jul 21, 2026, 6:07 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Remote Neurocognitive and Psychological Assessment in PKU

NCT04943393
View directory record Official record
Conditions
Phenylketonurias
Interventions
nointervention
Other
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 40 Years
Enrollment
84 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia • Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 21, 2022 · Synced Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 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 23, 2026 · Synced Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 PM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

NCT01212744
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
9
States / cities
Aurora, Colorado • Gainesville, Florida • Louisville, Kentucky + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2019 · Synced Jul 21, 2026, 6:07 PM EDT
Completed Phase 2 Interventional

Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria

NCT04534842
View directory record Official record
Conditions
Phenylketonuria
Interventions
SYNB1618, SYNB1934
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
11
States / cities
Aurora, Colorado • Boca Raton, Florida • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2022 · Synced Jul 21, 2026, 6:07 PM EDT
Recruiting Phase 3 Interventional

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT06302348
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sepiapterin
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 9 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 21, 2026, 6:07 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 Jun 22, 2026 · Synced Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 PM 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 28, 2023 · Synced Jul 21, 2026, 6:07 PM 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 Dec 1, 2025 · Synced Jul 21, 2026, 6:07 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 18, 2018 · Synced Jul 21, 2026, 6:07 PM 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 Jan 31, 2016 · Synced Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 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 5, 2019 · Synced Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 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 Jul 21, 2026, 6:07 PM EDT