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A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease

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Trial Profile

A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 13 Feb 2026

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At a glance

  • Drugs Cipaglucosidase alfa (Primary) ; Miglustat (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions; Registrational
  • Acronyms AMICUS ATB200-07
  • Sponsors Amicus Therapeutics

Most Recent Events

  • 03 Feb 2026 According to an Amicus Therapeutics media release, data from this trial are being presented at the 22nd Annual WORLDSymposium 2026
  • 19 Mar 2025 Status changed from active, no longer recruiting to completed.
  • 01 May 2024 Results (n=118) published in the Journal of Neurology

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