Phenylketonuria — Trials, FDA Actions & Pivotal Research

10 active Phenylketonuria trials currently recruiting in the US, sourced from ClinicalTrials.gov.

Synced daily from openFDA and ClinicalTrials.gov. Last sync: June 12, 2026.

Recruiting trials by city

Cities where Phenylketonuria trials in our archive had recruiting sites. Each link lists every currently recruiting paid clinical trial at that location.

Los Angeles, CA Boston, MA Indianapolis, IN

Active Phenylketonuria trials

Currently recruiting US trials matching Phenylketonuria, sourced live from ClinicalTrials.gov. We’ll add per-event articles here as those trials post results or hit primary completion.

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

for Phenylketonuria

1 location in Dallas

Sponsor: Agios Pharmaceuticals, Inc.

Sex: All·Age: 18 - 69

Code: NCT07241234·Phase 1·Recruiting

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Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

for Phenylketonuria

12 locations incl. Los Angeles

Sponsor: BioMarin Pharmaceutical

Sex: All·Age: 18 - 65

Code: NCT07477691·Phase 4·Not Yet Recruiting

PKU Protein Requirements

for PKU

1 location in Atlanta

Sponsor: Emory University

Sex: All·Age: 18+

Code: NCT07194993·Recruiting

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A Study of JNT-517 in Participants With Phenylketonuria (PKU)

for Phenylketonuria

10 locations incl. Los Angeles

Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.

Sex: All·Age: 18+

Code: NCT06971731·Phase 3·Recruiting

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Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in Classical Phenylketonuria Patients Undergoing Aerobic and Resistance Exercises

for Phenylketonuria

Sponsor: Hacettepe University

Sex: All·Age: 18+

Code: NCT07526909·Recruiting

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GMP Powdered Substitutes in PKU and TYR

for Phenylketonuria

Sponsor: Nutricia UK Ltd

Sex: All·Age: 3+

Code: NCT06941532·Recruiting

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Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience.

for Phenylketonuria

Sponsor: Assistance Publique - Hôpitaux de Paris

Sex: All·Age: 18+

Code: NCT06289348·Not Yet Recruiting

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

for Phenylketonuria

1 location in Indianapolis

Sponsor: PTC Therapeutics

Sex: All·Age: Up to 9

Code: NCT06302348·Phase 3·Recruiting

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

for Phenylketonuria

2 locations incl. Boston

Sponsor: Homology Medicines, Inc

Sex: All·Age: 18 - 55

Code: NCT04348708·Enrolling By Invitation

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

for Pregnancy

1 location in Atlanta

Sponsor: Emory University

Sex: Female·Age: 11+·Accepts healthy volunteers

Code: NCT01659749·Recruiting

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