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Gene Therapy for FHL3 Using Transduced Autologous CD34+ Cells
NCT06736080Phase 2Not yet recruiting
- Sponsor
- Assistance publique - Hôpitaux de Paris
- Start
- Jan 2025
- Primary completion
- Jan 2029
- Modality
- Gene Editing, Gene Silencing, Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of MUNC13.4-CD34 in familial hemophagocytic lymphohistiocytosis type 3 (FHL3), sponsored by Assistance publique - Hôpitaux de Paris. Primary endpoints: Transplantation Related Mortality at 6 months, Adverse Events at 60 months and 2 more. Planned start Jan 2025; status: not yet recruiting.
02
Drugs
MUNC13.4-CD34
03
Conditions
familial hemophagocytic lymphohistiocytosis type 3 (FHL3)
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