Fabry disease patients monitored for Real-World treatment effects

NCT ID NCT06906367

Recruiting now Knowledge-focused Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jul 10, 2026 · Updated 2 times

Summary

This study follows 450 US adults with Fabry disease who are taking migalastat or enzyme replacement therapy. Researchers will track kidney function, heart and brain events, and quality of life over time to see how well these treatments work in everyday practice.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: migalastat HCl and enzyme replacement therapy (ERT)
What this could lead to: If successful, this study could provide real-world evidence on how well migalastat works over time for Fabry disease, potentially guiding better treatment decisions.
What could go wrong: This is an observational study, not a controlled trial, so results may be influenced by patient differences. It does not test a new treatment, only monitors existing ones.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Fabry disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FABRY DISEASE

Contacts and locations

Locations

Arkansas Children's Hospital
RECRUITING

Little Rock, Arkansas, 72202, United States
Emory Genetics
RECRUITING

Atlanta, Georgia, 30322, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
RECRUITING

Fairfax, Virginia, 22030, United States
New York-Presbyterian Morgan Stanley Children's Hospital - Columbia University Medical Center
NOT_YET_RECRUITING

New York, New York, 10032, United States
Renal Disease Research Institute
RECRUITING

Dallas, Texas, 75204, United States
UAB Nephrology Research Clinic at Paula Building
RECRUITING

Birmingham, Alabama, 35233, United States
UPMC Children's Hospital of Pittsburgh
NOT_YET_RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
Washington University School of Medicine
RECRUITING

St Louis, Missouri, 63110, United States

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