Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)

PHASE1/PHASE2RecruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

July 19, 2023

Primary Completion Date

July 19, 2041

Study Completion Date

July 19, 2041

Conditions

Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency

Interventions

GENETIC

ARTEGENE drug product

Each patient will receive a single intravenous infusion of ARTEGENE drug product at D0.

Trial Locations (1)

RECRUITING

Department of Pediatric Immunology, Hematology and Rheumatology UIHR, Necker-Enfants Malades Hospital, Paris

All Listed Sponsors

lead

Assistance Publique - Hôpitaux de Paris

OTHER