Safety and Efficacy of Gene Therapy of FHL Type 3 Caused by Mutations in the Human UNC13D Gene by Transplantation of a Single Dose of Autologous CD34+ Cells Transduced ex Vivo With the UNC13D LV Vector Expressing the UNC13D cDNA

Clinical trial NCT06736080 · NOT_YET_RECRUITING

Trial details

NCT number: NCT06736080
Sponsor: Assistance Publique - Hôpitaux de Paris
Status: NOT_YET_RECRUITING
Phase: Phase 1, Phase 2
Study type: INTERVENTIONAL

Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3)

Start date: 2026-05-01
Primary completion: 2029-01-01
Enrollment: 5

View on ClinicalTrials.gov

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