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Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

NCT07445490 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 6

Last updated 2026-03-06

No results posted yet for this study

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.

Conditions

  • Tay-Sachs Disease Ganglioside
  • Sandhoff Disease Ganglioside

Interventions

BIOLOGICAL

blood sample

collecting blood sample for various analyses

Sponsors & Collaborators

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-31
Primary Completion
2026-05-31
Completion
2027-11-30

Countries

  • France

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 NCT07445490 on ClinicalTrials.gov