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NOT YET RECRUITING
NCT07445490
Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis
Sponsor: Assistance Publique - Hôpitaux de Paris
View on ClinicalTrials.gov
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.
Key Details
Gender
All
Age Range
5 Years - Any
Study Type
OBSERVATIONAL
Enrollment
6
Start Date
2026-05
Completion Date
2027-11
Last Updated
2026-03-06
Healthy Volunteers
No
Interventions
BIOLOGICAL
blood sample
collecting blood sample for various analyses
Locations (1)
Département de Neurologie
Paris, France