Clinical Trial

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

Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.
Protocol Amendment History 10 amendments
This ClinicalTrials.gov record has been amended 10 times since 2025-03-24; most recent amendment 2026-06-23.
Status change: Not Yet Recruiting → Recruiting 2025-06-05
Trial Details
NCT Number NCT06904261
Lead Sponsor Amicus Therapeutics
Conditions Fabry Disease
Enrollment 8 participants
Start Date 2026-01-08
Primary Completion 2027-12 (estimated)
Study Completion 2028-12 (estimated)
Updated on ClinicalTrials.gov 2026-06-25