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A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo

Trial Profile

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 31 May 2024

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At a glance

  • Drugs Cipaglucosidase alfa (Primary) ; Miglustat (Primary) ; Alglucosidase alfa
  • Indications Glycogen storage disease type II
  • Focus Registrational; Therapeutic Use
  • Acronyms PROPEL
  • Sponsors Amicus Therapeutics

Most Recent Events

  • 18 Apr 2024 Results assessing Effect Size Analysis of Cipaglucosidase Alfa Plus Miglustat Versus Alglucosidase Alfa in ERT-experienced Adults with Late-onset Pompe Disease presented at the 76th Annual Meeting of the American Academy of Neurology 2024
  • 01 Feb 2024 According to an Amicus Therapeutics media release, data from this study will be presented at the 20th Annual WORLDSymposium™ 2024, being held February 4-9, 2024 in San Diego, CA.
  • 28 Sep 2023 According to an Amicus Therapeutics media release, announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules for adults living with late-onset Pompe disease (LOPD).The FDA approval was based on clinical data observed from this trial.

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