NCT04049760

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 12 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects \> 12 Years of Age With Fabry Disease and Amenable GLA Variants

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	migalastat HCl 150 mg	migalastat HCl 150 mg capsule

Timeline

• Start date: 2019-10-11
• Primary completion: 2024-11-29
• Completion: 2024-11-29
• First posted: 2019-08-08
• Last updated: 2026-02-05
• Results posted: 2025-06-17

Locations

6 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study