Testing effectiveness (Phase 2)Not Yet RecruitingNCT06736080
What this trial is testing
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
Who this might be right for
Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3)
Assistance Publique - Hôpitaux de Paris 5