NCT04798235View on ClinicalTrials.gov

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

3

Participants

Timeline

Start Date

March 12, 2021

Primary Completion Date

March 12, 2027

Study Completion Date

March 12, 2027

Conditions
Infantile GM2 Gangliosidosis (Disorder)
Interventions
BIOLOGICAL

TSHA-101

AAV9 viral vector containing HEXA and HEXB genes to be administered via Intrathecal injection

Trial Locations (1)

K7L 2V7

Queen's University/Kingston Health Sciences Centre, Kingston

Sponsors

Collaborators (1)

GlycoNet
All Listed Sponsors
collaborator

Taysha Gene Therapies, Inc.

INDUSTRY

collaborator

GlycoNet

UNKNOWN

lead

Dr. Anupam Sehgal

OTHER