Not Yet RecruitingNot Applicable

Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

France6 participantsStarted 2026-05

Plain-language 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.

Who can participate

Age range5 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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 Exclusion Criteria: * 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

What they're measuring

Demonstrate effective cross-correction between myeloid cell lines (derived from patients) that have undergone ex vivo gene therapy and in vitro neurons derived from iPSCs from patients with GM2 gangliosidosis.

Timeframe: 18 months

Trial details

NCT IDNCT07445490

SponsorAssistance Publique - Hôpitaux de Paris

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2026-05

Contact for this trial

Yann NADJAR, MD

01 42 16 17 52 yann.nadjar@aphp.fr

View on ClinicalTrials.gov More Tay-Sachs Disease Ganglioside trials