Active Not Recruiting
PHASE1, PHASE2
INTERVENTIONAL
NCT04798235
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
View on ClinicalTrials.gov
Updated Dec 15, 2025
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 lysosomes.Hex A is composed of 2 subunits, α- and β-, coded by the HEXA and HEXB genes, respectively. The primary purpose of the current study is to assess the safety and tolerability of TSHA101 administered via IT injection.
Interventions
Name:
TSHA-101
Type:
BIOLOGICAL
Description:
AAV9 viral vector containing HEXA and HEXB genes to be administered via Intrathecal injection
Primary Outcomes
Measure:
Safety and tolerability: Treatment-emergent Adverse Events (TEAEs)
TimeFrame:
1 year
Description:
Incidence, severity, and relatedness of TEAEs
Measure:
Safety and Tolerability: Number of participants with abnormal Laboratory assessments
TimeFrame:
1 year
Description:
Number of participants with Changes from Baseline in laboratory assessments
Measure:
Safety and Tolerability: Electrocardiogram (ECG)
TimeFrame:
1 year
Description:
Changes from Baseline in 12-lead ECG findings in QT interval
Trial Information
NCT ID
NCT04798235
Status
Active Not Recruiting
Study Type
INTERVENTIONAL
Phases
PHASE1, PHASE2
Sponsor
Dr. Anupam Sehgal
Last Updated
December 15, 2025