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

Join a study on Migalastat for Fabry disease with kidney issues.

Recruiting
18 years and older
All
Phase 3

This study looks at a medicine called migalastat for people with Fabry disease and kidney problems. Fabry disease is a condition caused by changes in a gene called GLA. The study focuses on people with severe kidney issues, where the kidneys can't filter blood well, measured by something called eGFR. Two groups are part of this study: one group with serious kidney issues not on dialysis, and another group needing regular dialysis (a process that removes waste from the blood).

Participants will have a screening visit to see if they can join. Eligible participants will have a second visit within 30 days. The study is for people 18 or older, with a specific type of Fabry disease that responds to migalastat. Participants should not have had kidney transplants or certain other treatments recently.

  • The study involves multiple visits and regular dialysis for some participants.
  • Participants must be willing to use birth control if they can have children.
  • People with certain health conditions or treatments can't join.
Study details
    Fabry Disease

NCT04020055

Amicus Therapeutics

12 April 2025

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

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Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

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The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

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Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

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Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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