Clinical Trial

Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

Study acronym: GM2-TGEX
Not Yet Recruiting
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Record status
This record was last updated March 6, 2026 (before its estimated May 2026 completion). Its status may not reflect the trial's current state.
Summary
The project aims to optimize and validate this new therapeutic strategy using cells from GM2 patients to evaluate the cross-correction of neurons in vitro by the culture medium of genetically modified myeloid cell lines. The ultimate goal is to demonstrate the potential of CHS-TGEX as an effective treatment in humans for GM2 gangliosidosis.
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2026-02-25.
Trial Details
NCT Number NCT07445490
Lead Sponsor Assistance Publique - Hôpitaux de Paris
Conditions Tay-Sachs Disease Ganglioside, Sandhoff Disease Ganglioside
Enrollment 6 participants
Start Date 2026-05
Primary Completion 2026-05 (estimated)
Study Completion 2027-11 (estimated)
Updated on ClinicalTrials.gov 2026-03-06