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

NCT04798235 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2023-05-09

No results posted yet for this study

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.

Conditions

  • Infantile GM2 Gangliosidosis (Disorder)

Interventions

BIOLOGICAL

TSHA-101

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

Sponsors & Collaborators

  • Taysha Gene Therapies, Inc.

    collaborator INDUSTRY

  • GlycoNet

    collaborator UNKNOWN

  • Dr. Anupam Sehgal

    lead OTHER

Principal Investigators

  • Anupam Sehgal, MBBS · Queen's University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
15 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-12
Primary Completion
2027-03-12
Completion
2027-03-12

Countries

  • Canada

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04798235 on ClinicalTrials.gov