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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
Flotte, Terence
NCT04669535: A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease	Completed	1	9	US	AXO-AAV-GM2 Starting Dose, AAVrh8-HEXA and AAVrh8-HEXB, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose, AXO-AAV-GM2 High Dose	Terence Flotte, University of Massachusetts, Worcester, Massachusetts General Hospital	Tay-Sachs Disease, Sandhoff Disease	05/24	12/24
NCT06614569: Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease	Active, not recruiting	N/A	7	US	AXO-AAV-GM2	Terence Flotte	GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease	12/28	12/28