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A Study to Evaluate Migalastat in Fabry Subjects with Amenable GLA Variant and Renal Disease

Testing Migalastat for Fabry patients with specific kidney-related gene changes.

Recruiting
18 years and older
All
Phase 3

This study is looking at how safe a drug called migalastat is for people with Fabry disease who also have kidney problems. Fabry disease is a genetic condition that affects the body's ability to break down a certain fat, leading to health problems. The study includes people whose kidneys are not working well (eGFR < 30 mL/min/1.73 m²), a measure of kidney function. Some participants may be on dialysis, which is a treatment that helps clean the blood when kidneys can’t.

There are two groups in this study:

  • Group 1: Patients with severe kidney problems not on dialysis.
  • Group 2: Patients with end-stage kidney disease who are on specific types of dialysis.

To join, participants must be 18 or older, have a type of Fabry disease that responds to migalastat, and have a low eGFR. They should not have had a kidney transplant or be on other specific drugs.

  • Duration: Screening followed by study visits over several weeks.
  • Location: Study clinic or affiliated dialysis center.
  • Eligibility: Must meet specific health criteria and commit to study requirements.
Study details
    Fabry Disease

NCT04020055

Amicus Therapeutics

30 March 2025

Step 1 Get in touch with the nearest study center
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