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Phenylketonuria (pku) Clinical Trials

6 recruiting trials for Phenylketonuria (pku). 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
1
Phase 3 Trials
4
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.

RECRUITINGNCT06305234

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

This is a 10-year multi-center, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq...

Sponsor: BioMarin PharmaceuticalEnrolling: 20011 locations
RECRUITINGNCT07484945

Multiomics Approach in Adult Patients With Phenylketonuria

The GENOPHEN study aims to explore the links between the genome, metabolomic profile, and clinical phenotype in adults with early-treated PKU.

Sponsor: University Hospital, ToursEnrolling: 14915 locations
RECRUITINGNCT06969209

Brain Aging in Phenylketonuria

Background: Historically, the primary goal in managing phenylketonuria (PKU) has been to prevent severe and irreversible intellectual disability, as well as to address nutritional...

Sponsor: Insel Gruppe AG, University Hospital BernEnrolling: 901 location
RECRUITINGPhase 2NCT06637514

A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria

The goal of this Phase 2, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adolescents (12 to less than 18 years of age) with...

Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.Enrolling: 107 locations
RECRUITINGPhase 3NCT06628128

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

The goal of this Phase 3, open-label study is to evaluate the long-term safety of JNT-517 in pediatric and adult participants with Phenylketonuria (PKU) after completion of either...

Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.Enrolling: 24012 locations
RECRUITINGNCT05813678

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

This is a 10-year multi-center, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and...

Sponsor: BioMarin PharmaceuticalEnrolling: 45020 locations

Frequently Asked Questions

There are currently 6 clinical trials for Phenylketonuria (pku), 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 (pku), 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 1 Phase 3 trials for Phenylketonuria (pku), 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.