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A Two-Stage, Dose-Escalation and Safety & Efficacy Study of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

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Trial Profile

A Two-Stage, Dose-Escalation and Safety & Efficacy Study of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 13 Jan 2025

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At a glance

  • Drugs Cobnabexagene anvuparvovec (Primary)
  • Indications Sandhoff disease; Tay-Sachs disease
  • Focus Adverse reactions
  • Sponsors Sio Gene Therapies [CEASED]

    • Most Recent Events

    • 09 Jan 2025 Status changed from active, no longer recruiting to completed.
    • 13 Dec 2023 Status changed from recruiting to active, no longer recruiting.
    • 21 Jul 2023 Planned number of patients changed from 18 to 10.

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