A Study of Patients With Fabry Disease (US Specific)

Recruiting now

Conditions studied: Fabry Disease

In brief

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.

Key facts

Study ID

NCT06906367

Run by

Amicus Therapeutics

People needed

450

Starts

2026-02-13

Expected to finish

2032-06-01

Last updated by the study team

2026-07-10

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Patients who currently are participating in a clinical trial of any investigational medicinal product or device at the time of enrollment

Where it is running

• UAB Nephrology Research Clinic at Paula Building — Birmingham, Alabama, United States (enrolling)
• Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
• Emory Genetics — Atlanta, Georgia, United States (enrolling)
• Washington University School of Medicine — St Louis, Missouri, United States (enrolling)
• Renal Disease Research Institute — Dallas, Texas, United States (enrolling)
• Lysosomal and Rare Disorders Research and Treatment Center, Inc. — Fairfax, Virginia, United States (enrolling)
• New York-Presbyterian Morgan Stanley Children's Hospital - Columbia University Medical Center — New York, New York, United States
• UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States

Full record on ClinicalTrials.gov

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