🏛️ Verified NIH Record • Updated 2026-07-09
A Study of Patients With Fabry Disease (US Specific) (NCT06906367)
🟢 Recruiting Interventional Protocol 📊 Observational Clinical Research Sponsor: Amicus Therapeutics
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: UAB Nephrology Research Clinic at Paula Building — Birmingham, Alabama
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (8 Sites)
- UAB Nephrology Research Clinic at Paula Building — Birmingham, Alabama
- Arkansas Children's Hospital — Little Rock, Arkansas
- Emory Genetics — Atlanta, Georgia
- Washington University School of Medicine — St Louis, Missouri
- New York-Presbyterian Morgan Stanley Children's Hospital - Columbia University Medical Center — New York, New York
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania
- Renal Disease Research Institute — Dallas, Texas
- Lysosomal and Rare Disorders Research and Treatment Center, Inc. — Fairfax, Virginia
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.