A Phase 3b Open-Label Study of 12-Month Migalastat Treatment in Children Aged 2 to Under 12 Years with Fabry Disease and Amenable GLA Variants

What this Trial Is About

Researchers are evaluating the safety, pharmacokinetics PK, pharmacodynamics PD, and efficacy of migalastat treatment in children aged 2 to less than 12 years with Fabry disease who have specific amenable GLA gene variants. This Phase 3b, open-label, uncontrolled, multicenter study focuses on pediatric subjects who have either never received enzyme replacement therapy ERT or have stopped ERT at least 14 days before the baseline visit. Participants will receive migalastat orally every other day, with the initial dose based on body weight at baseline. The study includes two consecutive treatment stages Stage 1 lasts about 3 months 12 weeks, followed immediately by Stage 2 lasting 9 months. After completing treatment, participants who stop treatment will have a 30-day safety follow-up period. Blood samples will be collected at specific times to measure migalastat levels and understand how the drug behaves in the body. During the study, participants will be monitored through various assessments including laboratory tests to evaluate safety, kidney function, heart measurements, symptom scores, and quality of life questionnaires. Blood samples for PK analysis will be collected at multiple points including during the first month and at 6 and 12 months. The study will track side effects and changes in disease markers throughout the 12 months of treatment, with continued safety monitoring during the follow-up period.

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