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Clinical Trials in France / NCT07445490
Starting soon Observational

Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

NCT07445490 · tracked via the Priya Life Science France tracker
Sponsor
Assistance Publique - Hôpitaux de Paris
Phase
Observational
Started
2026-05
Last updated
2026-03-06

Condition(s) studied

Tay-Sachs Disease GangliosideSandhoff Disease Ganglioside

Investigational drug(s) / intervention(s)

blood sample →

blood sample: collecting blood sample for various analyses

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.

Eligibility

Sex
ALL
Min age
5 Years
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

  • 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. — 18 months
    100% increase in neuronal β-hexosaminidase enzyme activity after cross-correction.

Trial sites (1)

FacilityCityRegionStatus
Département de Neurologie Paris France

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07445490 on ClinicalTrials.gov ↗ ← All trials in France