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

NCT06736080 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

NOT_YET_RECRUITING

Status

5

Enrollment

OTHER

Sponsor class

Conditions

Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3)

Interventions

DRUG: MUNC-CD34
DRUG: MUNC-T3

Sponsor

Assistance Publique - Hôpitaux de Paris