Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

Starting soon

Conditions studied: Tay-Sachs Disease Ganglioside, Sandhoff Disease Ganglioside

In brief

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 facts

Study ID

NCT07445490

Run by

Assistance Publique - Hôpitaux de Paris

People needed

6

Starts

2026-05-01

Expected to finish

2027-11-01

Last updated by the study team

2026-03-06

Who can join

Age: 5 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Proven diagnosis of GM2 gangliosidosis (decreased β-hexosaminidase enzyme activity and/or biallelic pathogenic variants in the HEXA or HEXB gene)
• Age ≥ 5 years
• Blood sample planned as part of treatment

You may not qualify if…

• Opposition from the patient or legal guardians
• Contraindication to venous sampling
• Patient under guardianship or curatorship
• Patient not covered by social security
• Patient covered by AME (State Medical Aid)
• Weight < 25 kg for minor patients

Where it is running

• Département de Neurologie — Paris, France

Full record on ClinicalTrials.gov

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