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Clinical trial

Long-Term Follow-Up of A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

NCT06614569 is registered in ClinicalTrials.gov with Terence Flotte as lead sponsor.

NCT NCT06614569
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Registry record

As filed in ClinicalTrials.gov.

Title
Long-Term Follow-Up of A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
Lead sponsor
Terence Flotte
Phase
Observational
Overall status
Active, not recruiting
Start date
September 17, 2024
Completion date
December 31, 2028
Enrollment
7 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

gm2 gangliosidosis, sandhoff disease, tay sachs disease