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A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe Disease

Trial Profile

A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe Disease

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 28 Feb 2024

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At a glance

  • Drugs Zocaglusagene nuzaparvovec (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions; First in man; Pharmacodynamics
  • Acronyms FORTIS
  • Sponsors Astellas Gene Therapies
  • Most Recent Events

    • 22 Feb 2024 Planned End Date changed from 30 Apr 2029 to 31 Aug 2029.
    • 22 Feb 2024 Planned primary completion date changed from 30 Apr 2029 to 31 Aug 2029.
    • 05 Dec 2023 Planned End Date changed from 31 May 2029 to 30 Apr 2029.
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