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Clinical trial

Phase 1/2, Open-Label Clinical Study to Evaluate the Safety and Efficacy of Intrathecal TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

NCT04798235 is registered in ClinicalTrials.gov with Dr. Anupam Sehgal as lead sponsor.

NCT NCT04798235
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Registry record

As filed in ClinicalTrials.gov.

Title
Phase 1/2, Open-Label Clinical Study to Evaluate the Safety and Efficacy of Intrathecal TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
Lead sponsor
Dr. Anupam Sehgal
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
March 12, 2021
Completion date
March 12, 2027
Enrollment
3 (actual)
Sites
1

Conditions

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

infantile gm2 gangliosidosis (disorder)