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An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants

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Trial Profile

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 17 Jun 2025

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At a glance

  • Drugs Migalastat (Primary)
  • Indications Fabry's disease
  • Focus Adverse reactions; Pharmacokinetics
  • Sponsors Amicus Therapeutics

Most Recent Events

  • 10 Jun 2025 Planned initiation date changed from 1 May 2025 to 1 Jun 2025.
  • 10 Jun 2025 Status changed from not yet recruiting to recruiting.
  • 04 Apr 2025 New trial record

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