Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for Fabry Disease Clinical Trials
Fabry Disease clinical trials are recruiting across 26 cities. Here are the cities with the most active studies:
Fabry Disease Trial Phases
6 research organizations are sponsoring fabry disease trials.
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, 10 studies are recruiting a combined 9,690 participants across the United States. Research is being conducted by 6 organizations including Genzyme, a Sanofi Company, Amicus Therapeutics, Chiesi Farmaceutici S.p.A. and 3 others.
2026 Fabry Disease Research Landscape
As of July 2026, the fabry disease clinical trial landscape includes 10 actively recruiting studies across 26 cities in the United States. These studies are collectively seeking 9,690 participants, with an average enrollment target of 969 per study.
The research spans multiple phases of development: 3 studies are in PHASE2, 2 studies are in PHASE3, 2 studies are in PHASE1. The presence of Phase 3 trials indicates that some fabry disease treatments are in late-stage testing and may be approaching regulatory review.
Research is being led by 6 different organizations, including Genzyme, a Sanofi Company, Amicus Therapeutics, Chiesi Farmaceutici S.p.A., University of Minnesota, GC Biopharma Corp, and 1 others.
Geographically, fabry disease trials are most concentrated in Fairfax, Virginia (6 trials); Atlanta, Georgia (5 trials); Birmingham, Alabama (4 trials); Pittsburgh, Pennsylvania (4 trials); Minneapolis, Minnesota (4 trials) and 7 other cities.
Featured Fabry Disease Studies
Highlighted recruiting studies for fabry disease, selected by enrollment size and research scope.
Fabry Disease Registry & Pregnancy Sub-registry
The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The primary objectives of the Reg...
A Study of Patients With Fabry Disease (US Specific)
This is an observational study to evaluate the effects of treatment on long-term effectiveness, safety, and health-related quality of life (HRQOL) in patients with Fabry disease, with a main focus on migalastat.
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.
Frequently Asked Questions About Fabry Disease Clinical Trials
Are there fabry disease clinical trials near me?
Yes, there are 10 fabry disease clinical trials currently recruiting across 26+ cities in the United States, including Fairfax, Virginia; Atlanta, Georgia; Birmingham, Alabama. 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 across PHASE2 (3 studies), PHASE3 (2 studies), PHASE1 (2 studies). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 6 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 July 17, 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 17, 2026 · Data from ClinicalTrials.gov