Phenylketonuria Clinical Trials
8 recruiting trials for Phenylketonuria. Eligibility criteria explained in plain English.
TrialFinderData lists 8 Phenylketonuria clinical trials drawn from ClinicalTrials.gov, all of which are currently recruiting participants.
Across the trials that carry a phase, Phase 3 is the largest group at 50% (2 studies); the largest phase groups are Phase 3: 2, Phase 4: 1, Phase 1: 1.
Research is led by PTC Therapeutics (1), BioMarin Pharmaceutical (1), Emory University (1), among the most active sponsors registered for these trials.
The most frequently studied intervention is JNT-517 Tablet (drug, 2 trials), followed by Sepiapterin, Pegvaliase.
Track Phenylketonuria trials
Subscribe for TrialFinderData updates by email. No spam, unsubscribe anytime.
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 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...
Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
Study 165-401 is a Phase 4, open-label study designed to examine the concomitant use of methotrexate (MTX) to suppress immune responses to Palynziq and improve tolerability and...
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...
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...
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).
Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience.
The aims of this collaborative, interdisciplinary research project are to understand and describe the psychological impact of the announcement of a rare, serious disease present...
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...
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...
Explore Other Conditions
Frequently Asked Questions
There are currently 8 clinical trials for Phenylketonuria, with 8 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.
Trial data sourced from the ClinicalTrials.gov API. This site does not provide medical advice, always talk to your doctor about clinical trial participation.