Skip to main content
TTrialFinderData
TrialFinderData is for informational purposes only and does not provide medical advice. Always talk to your doctor.

Phenylketonuria Clinical Trials

6 recruiting trials for Phenylketonuria. Eligibility criteria explained in plain English.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.
6
Total Trials
6
Recruiting Now
2
Phase 3 Trials
6
Sponsors

Recruiting Trials

Clinical trial data sourced from the ClinicalTrials.gov registry, maintained by the National Library of Medicine. Always consult your doctor before considering any clinical trial.

RECRUITINGPhase 1NCT07241234

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

The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).

Sponsor: Agios Pharmaceuticals, Inc.Enrolling: 201 location
RECRUITINGPhase 3NCT06302348

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in...

Sponsor: PTC TherapeuticsEnrolling: 568 locations
RECRUITINGNCT01659749

Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and...

The purpose of this project is to study the effectiveness of teaching teens and young women with Phenylketonuria (PKU) or Maple Syrup Urine Disease (MSUD) about their disease and...

Sponsor: Emory UniversityEnrolling: 2001 location
RECRUITINGNCT06941532

GMP Powdered Substitutes in PKU and TYR

Four new GMP-based protein substitutes have been developed to support the dietary management of PKU and TYR. These products are powdered protein substitutes, low in phenylalanine...

Sponsor: Nutricia UK LtdEnrolling: 401 location
RECRUITINGNCT07526909

Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in...

This study will be the first in the literature to evaluate the relationship between aerobic and resistance exercises, plasma amino acid levels, metabolic control of patients, and...

Sponsor: Hacettepe UniversityEnrolling: 101 location
RECRUITINGPhase 3NCT06971731

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

The goal of this Phase 3, randomized study is to assess the safety, efficacy, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adults (18 years of age or older) with...

Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.Enrolling: 12020 locations

Frequently Asked Questions

There are currently 6 clinical trials for Phenylketonuria, with 6 actively recruiting participants. These include trials across all phases from early-stage Phase 1 to late-stage Phase 3.

To join a clinical trial for Phenylketonuria, review the eligibility criteria on the trial detail pages, then talk to your doctor about whether a trial is right for you. Your doctor can help you evaluate the potential benefits and risks.

Phase 3 trials are large-scale studies that test whether a treatment is effective and monitor side effects. There are 2 Phase 3 trials for Phenylketonuria, representing treatments closest to potential FDA approval.

Clinical trials follow strict safety protocols overseen by Institutional Review Boards (IRBs) and the FDA. Participants are monitored closely and can withdraw at any time. Always discuss risks and benefits with your healthcare provider before enrolling.

Sources: ClinicalTrials.gov, FDA
Last updated:

Trial data sourced from the ClinicalTrials.gov API. This site does not provide medical advice, always talk to your doctor about clinical trial participation.

this entity is one of the data points covered by this site’s U.S. clinical trials and research registries dataset. The detail above comes directly from the NIH ClinicalTrials.gov registry; the context that follows situates the headline numbers against the broader distribution across active and historical clinical trials.

Every number on this page links back to the NIH ClinicalTrials.gov registry; the methodology page describes the inputs, refresh cadence, and known limitations of the underlying data product.

For readers using this page as a decision input, the related-entity pages elsewhere on the site provide the comparison set. The most useful comparison for this entity is typically a peer within active and historical clinical trials with similar size, similar exposure, or similar geography — not the national-level summary alone.