Clinical trial
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
NCT04669535 is registered in ClinicalTrials.gov with Terence Flotte as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- 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
- Lead sponsor
- Terence Flotte
- Phase
- Phase 1
- Overall status
- Terminated
- Start date
- January 15, 2021
- Completion date
- December 16, 2024
- Enrollment
- 9 (actual)
- Sites
- 2
Conditions
As the registry lists them; a condition with its own page is linked.