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A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

Clinical trial for kids with Fabry disease and specific genetic variants.

Recruiting
2-11 years
All
Phase 3

This study tests the safety and effects of a drug called migalastat in children aged 2 to less than 12 years with Fabry disease, a rare genetic condition. Migalastat is used for children with specific changes in their GLA gene (a part of DNA) that the drug can help with. The study will last for 12 months. There are two main stages: the first is 3 months long, and the second is 9 months long. After these, there is an open-label extension, meaning everyone knows they're getting the drug.

During the study, children will have blood tests to check how the drug moves and works in their bodies. Children who have stopped or never started enzyme replacement therapy (ERT) can join, but those with serious kidney problems or allergies to the drug cannot. The study requires the child's parent or guardian to agree and sign a consent form.

  • Study Duration: 12 months with additional follow-up if needed.
  • Visits: Regular visits for check-ups and blood tests.
  • Participation: No recent ERT and specific gene types required.
Study details
    Fabry Disease

NCT06904261

Amicus Therapeutics

17 October 2026

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