1 active not recruiting, 1 terminated. 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Terminated 1 |
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease AXO-AAV-GM2 - GM2 Gangliosidosis, Tay Sachs Disease | Active not recruiting | Terence Flotte | 7 | 2024-09-17 | 2025-10-16 | |
| A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose - Tay-Sachs Disease, Sandhoff Disease | Phase 1 | Terminated | Terence Flotte | 9 | 2021-01-15 | 2025-09-02 |
Gangliosidoses, GM2 (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · Lysosomal Storage Diseases (1)
United States (2)