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An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response

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Trial Profile

An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response

Status: Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 30 Apr 2024

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At a glance

  • Drugs Avalglucosidase alfa (Primary) ; Alglucosidase alfa
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions
  • Acronyms Mini-COMET
  • Sponsors Sanofi Genzyme

    • Most Recent Events

    • 25 Apr 2024 According to a Sanofi media release, data from this study were featured at the 20th annual WORLDSymposium.
    • 06 Feb 2024 Results published in the Sanofi Media Release.
    • 05 Feb 2024 Results presented in a Sanofi Media Release.

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