Phenylketonuria (pku) Clinical Trials
5 recruiting trials for Phenylketonuria (pku). Eligibility criteria explained in plain English.
TrialFinderData lists 5 Phenylketonuria (pku) clinical trials drawn from ClinicalTrials.gov, all of which are currently recruiting participants.
Every phased trial in this set is Phase 3 (1 of the listed studies); the remaining records are observational or have no phase recorded on ClinicalTrials.gov.
Research is led by BioMarin Pharmaceutical (2), Otsuka Pharmaceutical Development & Commercialization, Inc. (1), Insel Gruppe AG, University Hospital Bern (1), among the most active sponsors registered for these trials.
The most frequently studied intervention is Pegvaliase (drug, 2 trials), followed by JNT-517.
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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.
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...
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...
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...
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...
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.
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Frequently Asked Questions
There are currently 5 clinical trials for Phenylketonuria (pku), with 5 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.
Trial data sourced from the ClinicalTrials.gov API. This site does not provide medical advice, always talk to your doctor about clinical trial participation.