A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease....
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....
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease ...
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...
Top Cities for Fabry Disease Clinical Trials
Fabry Disease clinical trials are recruiting across 30 cities. Here are the cities with the most active studies:
About Fabry Disease
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.
Clinical trials are advancing new treatments for fabry disease. Currently, 4 studies are recruiting a combined 66 participants across the United States. Research is being conducted by 4 organizations including Chiesi Farmaceutici S.p.A., GC Biopharma Corp, Amicus Therapeutics and 1 others.
2026 Fabry Disease Research Landscape
As of March 2026, the fabry disease clinical trial landscape includes 4 actively recruiting studies across 30 cities in the United States. These studies are collectively seeking 66 participants, with an average enrollment target of 17 per study.
Research is being led by 4 different organizations, including Chiesi Farmaceutici S.p.A., GC Biopharma Corp, Amicus Therapeutics, UniQure Biopharma B.V..
Geographically, fabry disease trials are most concentrated in Atlanta, Georgia (3 trials); Fairfax, Virginia (3 trials); Cincinnati, Ohio (2 trials); Salt Lake City, Utah (2 trials); Minneapolis, Minnesota (2 trials) and 7 other cities.
Featured Fabry Disease Studies
Highlighted recruiting studies for fabry disease, selected by enrollment size and research scope.
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
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.
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease (ESRD)
Frequently Asked Questions About Fabry Disease Clinical Trials
Are there fabry disease clinical trials near me?
Yes, there are 4 fabry disease clinical trials currently recruiting across 30+ cities in the United States, including Atlanta, Georgia; Fairfax, Virginia; Cincinnati, Ohio. Browse the studies above to find one at a location convenient for you.
How do I join a fabry disease clinical trial?
To join a fabry disease 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 fabry disease clinical trials free?
Yes, participation in fabry disease 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 fabry disease treatments are being studied?
Current fabry disease clinical trials are testing a range of approaches. These include new drugs, combination therapies, medical devices, and other interventions sponsored by 4 research organizations.
Is it safe to participate in fabry disease 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