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A Phase 1 Study of the Safety of AAV2/8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease

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A Phase 1 Study of the Safety of AAV2/8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease

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

Latest Information Update: 03 Jul 2023

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At a glance

  • Drugs ACTUS 101 (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions
  • Sponsors Actus Therapeutics; Asklepios BioPharmaceutical

    • Most Recent Events

    • 27 Jun 2023 Planned End Date changed from 1 Apr 2028 to 1 Mar 2026.
    • 27 Jun 2023 Status changed from recruiting to active, no longer recruiting.
    • 25 May 2022 According to ClinicalTrials.gov record, protocol has been amended to increase patient number from 8 to 13, treatment arm changed from 3 to 5. Primary endpoint changed from 52 weeks to 78 weeks, other endpoint also changed. Official title also changed

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