Actively Recruiting
A Study of Patients With Fabry Disease (US Specific)
Led by Amicus Therapeutics · Updated on 2026-04-28
450
Participants Needed
8
Research Sites
328 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study of Patients With Fabry Disease (US Specific)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults 18 years or older with Fabry disease and amenable GLA variants
- Migalastat-treated patients who started treatment within 24 months before enrollment with eGFR ≥ 30 mL/min/1.73 m2 and are currently taking migalastat
- Patients showing decline in Fabry disease symptoms including decreased eGFR, microalbuminuria/macroalbuminuria, proteinuria, or males with classic Fabry disease phenotype
- Migalastat-treated patients from prior clinical trials exposed to treatment for at least 24 months with eGFR ≥ 30 mL/min/1.73 m2 and still on migalastat
- Untreated patients with amenable GLA variants, eGFR ≥ 30 mL/min/1.73 m2, and meeting local treatment guidelines
- ERT-treated patients who started within 24 months before enrollment, with eGFR ≥ 30 mL/min/1.73 m2 and amenable GLA variants
- All participants must be able to understand and provide written informed consent or assent
You will not qualify if you...
- Patients currently participating in a clinical trial of any investigational medicinal product or device at the time of enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 8 locations
1
UAB Nephrology Research Clinic at Paula Building
Birmingham, Alabama, United States, 35233
Not Yet Recruiting
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
3
Emory Genetics
Atlanta, Georgia, United States, 30322
Actively Recruiting
4
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Not Yet Recruiting
5
New York-Presbyterian Morgan Stanley Children's Hospital - Columbia University Medical Center
New York, New York, United States, 10032
Not Yet Recruiting
6
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Not Yet Recruiting
7
Renal Disease Research Institute
Dallas, Texas, United States, 75204
Actively Recruiting
8
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia, United States, 22030
Actively Recruiting
Research Team
A
Amicus Therapeutics Patient Advocacy
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
3
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