Recruiting PKU Studies in Pittsburgh
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...
About PKU Clinical Trials in Pittsburgh
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.
There are currently 2 pku clinical trials recruiting participants in Pittsburgh, PENNSYLVANIA. These studies are seeking a combined 212 participants. Research is being sponsored by PTC Therapeutics, NGGT INC.. Clinical trial participation is free and participants receive study-related medical care at no cost.
PKU Clinical Trials in Pittsburgh — FAQ
Are there pku clinical trials in Pittsburgh?
Yes, there are 2 pku clinical trials currently recruiting in Pittsburgh, PENNSYLVANIA. Browse the studies on this page to find one that fits.
How do I join a clinical trial in Pittsburgh?
Browse the studies listed above, click one that interests you, and complete the free eligibility form. A study coordinator at the Pittsburgh research site will contact you about next steps.
Are clinical trials in Pittsburgh free?
Yes, clinical trial participation is always free. Study-related treatment, medical tests, and doctor visits are provided at no cost. Many Pittsburgh studies also compensate for your time and travel.
What pku treatments are being tested?
The 2 active trials in Pittsburgh are testing new therapies including novel drugs, biologics, and treatment approaches for pku.
Data updated March 2, 2026 from ClinicalTrials.gov