A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein cons...
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT00...
Top Cities for PKU Clinical Trials
PKU clinical trials are recruiting across 13 cities. Here are the cities with the most active studies:
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, 2 studies are recruiting a combined 212 participants across the United States. Research is being conducted by 2 organizations including PTC Therapeutics, NGGT INC..
2026 PKU Research Landscape
As of March 2026, the pku clinical trial landscape includes 2 actively recruiting studies across 13 cities in the United States. These studies are collectively seeking 212 participants, with an average enrollment target of 106 per study.
Research is being led by 2 different organizations, including PTC Therapeutics, NGGT INC..
Geographically, pku trials are most concentrated in Pittsburgh, Pennsylvania (2 trials); Aurora, Colorado (1 trial); Gainesville, Florida (1 trial); Indianapolis, Indiana (1 trial); Boston, Massachusetts (1 trial) and 7 other cities.
Featured PKU Studies
Highlighted recruiting studies for pku, selected by enrollment size and research scope.
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is an rAAV8 based vector carrying a functional copy of the human PAH gene. Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
Frequently Asked Questions About PKU Clinical Trials
Are there pku clinical trials near me?
Yes, there are 2 pku clinical trials currently recruiting across 13+ cities in the United States, including Pittsburgh, Pennsylvania; Aurora, Colorado; Gainesville, Florida. 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. These include new drugs, combination therapies, medical devices, and other interventions sponsored by 2 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 March 2, 2026 from ClinicalTrials.gov
About This Data
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 March 2, 2026 · Data from ClinicalTrials.gov