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AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
NCT04669535Phase 1Terminated
- Start
- Jan 2021
- Primary completion
- May 2024
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of AXO-AAV-GM2 in Tay-Sachs disease (TSD) and Sandhoff disease. Primary endpoint: Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events at 48 weeks. Started Jan 2021; status: terminated.
02
Drugs
AXO-AAV-GM2
03
Conditions
Tay-Sachs disease (TSD)Sandhoff disease
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