Recruiting Fabry Disease Studies in Pittsburgh
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
This Phase 1/2 first-in-human (FIH) study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of HM15421 in patients with FD....
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people wi...
About Fabry Disease Clinical Trials in Pittsburgh
Fabry disease is a rare genetic disorder caused by deficiency of the enzyme alpha-galactosidase A, leading to accumulation of a fatty substance in cells. It can affect the heart, kidneys, and nervous system. Treatment includes enzyme replacement therapy and chaperone therapy.
There are currently 2 fabry disease clinical trials recruiting participants in Pittsburgh, PENNSYLVANIA. These studies are seeking a combined 30 participants. Research is being sponsored by GC Biopharma Corp, UniQure Biopharma B.V.. Clinical trial participation is free and participants receive study-related medical care at no cost.
Fabry Disease Clinical Trials in Pittsburgh — FAQ
Are there fabry disease clinical trials in Pittsburgh?
Yes, there are 2 fabry disease 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 fabry disease treatments are being tested?
The 2 active trials in Pittsburgh are testing new therapies including novel drugs, biologics, and treatment approaches for fabry disease.
Data updated March 2, 2026 from ClinicalTrials.gov