Status: Not yet recruiting
Standard of Care Comparative Arm of Phase 1/2 Gene Therapy Trial DREPAMIR" in Severe Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of transplantation of gene modified autologous CD34+ cells in SCD patients within a therapeutic strategy that may include anti-inflammatory treatment as a pre-transplant treatment in case of severe inflammation detected at the inclusion analysis; the autologous CD34+ cell will be transduced by the bifunctional βAS3m/miR7m lentiviral vector expressing the therapeutical beta-globin, βAS3m, and the miRNA anti-HbS vs Standard Of Care (SOC).
Participants needed: 30
Trial details
Age: 12-35Biological sex: AllType: InterventionalSponsor: Assistance Publique - Hôpitaux de ParisUpdated: Aug 7, 2026Locations: 1
Eligibility criteria
Age 12 - 35 years [+6]
Existence of a matched sibling donor [+22]