Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for PKU Clinical Trials
PKU clinical trials are recruiting across 23 cities. Here are the cities with the most active studies:
PKU Trial Phases
8 research organizations are sponsoring pku trials.
About PKU
Phenylketonuria (PKU) is an inherited metabolic disorder that increases levels of the amino acid phenylalanine in the blood, which can cause intellectual disability if untreated. Early detection through newborn screening is essential. Treatment includes a strict low-phenylalanine diet and enzyme substitution therapy.
Clinical trials are advancing new treatments for pku. Currently, 14 studies are recruiting a combined 1,327 participants across the United States. Research is being conducted by 8 organizations including BioMarin Pharmaceutical, Otsuka Pharmaceutical Development & Commercialization, Inc., PTC Therapeutics and 5 others.
2026 PKU Research Landscape
As of July 2026, the pku clinical trial landscape includes 14 actively recruiting studies across 23 cities in the United States. These studies are collectively seeking 1,327 participants, with an average enrollment target of 95 per study.
The research spans multiple phases of development: 3 studies are in PHASE3, 3 studies are in NA, 2 studies are in PHASE2, 2 studies are in PHASE1, 1 study is in PHASE4. The presence of Phase 3 trials indicates that some pku treatments are in late-stage testing and may be approaching regulatory review.
Research is being led by 8 different organizations, including BioMarin Pharmaceutical, Otsuka Pharmaceutical Development & Commercialization, Inc., PTC Therapeutics, Emory University, Maze Therapeutics, and 3 others.
Geographically, pku trials are most concentrated in Dallas, Texas (7 trials); Indianapolis, Indiana (6 trials); Pittsburgh, Pennsylvania (6 trials); Portland, Oregon (5 trials); Atlanta, Georgia (4 trials) and 7 other cities.
Featured PKU Studies
Highlighted recruiting studies for pku, selected by enrollment size and research scope.
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 tolerability, and the effectiveness of the additional risk minimization measures (aRMMs) (European Union (EU) only) in subjects receiving pegvaliase for the treatment of PKU. Subjects for whom a clinical decision has been m...
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 Study JNT517-101 (NCT05781399) or JNT517-201 (NCT06637514) as well as participants who have not participated in a prior JNT-517 study. In this trial, all participants will receive JNT-517 using age- and weight-banded dos...
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 treatment in subjects with phenylketonuria (PKU) in the United States (US). Subjects in the US for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date o...
Frequently Asked Questions About PKU Clinical Trials
Are there pku clinical trials near me?
Yes, there are 14 pku clinical trials currently recruiting across 23+ cities in the United States, including Dallas, Texas; Indianapolis, Indiana; Pittsburgh, Pennsylvania. Browse the studies above to find one at a location convenient for you.
How do I join a pku clinical trial?
To join a pku clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are pku clinical trials free?
Yes, participation in pku clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of pku treatments are being studied?
Current pku clinical trials are testing a range of approaches across PHASE3 (3 studies), NA (3 studies), PHASE2 (2 studies), PHASE1 (2 studies), PHASE4 (1 study). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 8 research organizations.
Is it safe to participate in pku clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated July 16, 2026 from ClinicalTrials.gov
About This Data
Data: ClinicalTrials.gov · Source of record for eligibility and locations
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated July 16, 2026 · Data from ClinicalTrials.gov