RecruitingNCT06906367

A Study of Patients With Fabry Disease (US Specific)

A Prospective, Observational Study of Patients With Fabry Disease (US Specific)


Sponsor

Amicus Therapeutics

Enrollment

450 participants

Start Date

Feb 13, 2026

Study Type

OBSERVATIONAL

Conditions

Summary

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.


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This observational study is following patients with Fabry disease in the United States who are being treated with migalastat (a pill that helps the body's enzyme system work better) or enzyme replacement therapy, to track how well the treatments work over time in real-world settings. **You may be eligible if...** - You are 18 or older with a confirmed diagnosis of Fabry disease - You have an amenable genetic variant (your specific mutation responds to migalastat) and are taking or starting migalastat commercially, OR you are receiving enzyme replacement therapy (ERT) - Your kidney function is adequate (eGFR ≥ 30 mL/min/1.73 m²) - You have not previously participated in a migalastat clinical trial **You may NOT be eligible if...** - You do not have a confirmed Fabry disease diagnosis - Your kidney function is below the minimum threshold - You are enrolled in another interventional clinical trial Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGmigalastat HCl

Non-interventional study of participants receiving migalastat HCl 150 mg

DRUGERT

Non-interventional study of participants receiving enzyme replacement therapy


Locations(8)

UAB Nephrology Research Clinic at Paula Building

Birmingham, Alabama, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Emory Genetics

Atlanta, Georgia, United States

Washington University School of Medicine

St Louis, Missouri, United States

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

Renal Disease Research Institute

Dallas, Texas, United States

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia, United States

View Full Details on ClinicalTrials.gov

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NCT06906367


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