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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 12 to <18 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 12 to <18 Years) With Fabry Disease and Amenable GLA Variants

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 13 Apr 2022

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At a glance

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

    • Most Recent Events

    • 02 Aug 2021 According to an Amicus Therapeutics media release, the company announced the European Commission has approved Galafold (migalastat) for use in adolescents aged 12 to <16 years weighing = 45 kg with a confirmed diagnosis of Fabry disease and who have an amenable mutation. The extension of the indication was supported by 1-month interim safety and pharmacokinetics data from this study
    • 12 Feb 2021 Status changed from active, no longer recruiting to completed.
    • 10 Feb 2020 Status changed from recruiting to active, no longer recruiting.
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