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A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations

Trial Profile

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 24 Jun 2019

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At a glance

  • Drugs Migalastat (Primary)
  • Indications Fabry's disease
  • Focus Registrational; Therapeutic Use
  • Acronyms FACETS
  • Sponsors Amicus Therapeutics
  • Most Recent Events

    • 18 Jan 2019 According to an Amicus Therapeutics media release, post hoc analysis results of this study will be presented at the 15th Annual WORLDSymposium 2019.
    • 10 Aug 2018 Results published in the Amicus Therapeutics Media Release
    • 10 Aug 2018 According to Amicus Therapeutics media release, TextFDA Approves Galafold (migalastat) for the Treatment of Certain Adult Patients with Fabry Disease.The FDA approval was based on data from this study.
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