New hope for fabry patients: Long-Term drug safety confirmed

NCT ID NCT04049760

Completed Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety of a drug called migalastat in 16 people over age 12 with Fabry disease, a rare genetic disorder. Participants had already completed an earlier study and continued taking migalastat to see if it was safe and how it affected kidney function. The goal was to manage the disease over time, not to cure it.

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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

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States
Lysosomal & Rare Disorders Research & Treatment Center

Fairfax, Virginia, 22030, United States
Royal Free London NHS Foundation Trust

London, United Kingdom
The Emory Clinic

Atlanta, Georgia, 30322, United States
University of Minnesota Masonic Children's Hospital and Clinics

Minneapolis, Minnesota, 55454, United States
University of South Florida

Tampa, Florida, 33606, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.