Status:
ACTIVE_NOT_RECRUITING
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
Lead Sponsor:
Dr. Anupam Sehgal
Collaborating Sponsors:
Taysha Gene Therapies, Inc.
GlycoNet
Conditions:
Infantile GM2 Gangliosidosis (Disorder)
Eligibility:
All Genders
Up to 15 years
Phase:
PHASE1
PHASE2
Brief Summary
GM2 gangliosidoses are a group of autosomal recessive neurodegenerative diseases characterized by a deficiency of the Hex A enzyme to catabolize GM2, thereby causing GM2 accumulation within cellular l...
Eligibility Criteria
Inclusion
- Key
- male or female with age less than or equal to 15 months
- diagnosis of GM2 gangliosidosis with genetic and enzymatic documentation of infantile disease
- Key
Exclusion
- a second neurodevelopmental disorder independent of the HEXA or HEXB
- inability to tolerate sedation or intrathecal administration
- invasive ventilatory support
- concomitant illness, allergies or known hypersensitivity to the required immunosuppression regimen
Key Trial Info
Start Date :
March 12 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 12 2027
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT04798235
Start Date
March 12 2021
End Date
March 12 2027
Last Update
May 9 2023
Active Locations (1)
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1
Queen's University/Kingston Health Sciences Centre
Kingston, Ontario, Canada, K7L 2V7