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).

NCT Number	NCT07752043
Lead Sponsor	Assistance Publique - Hôpitaux de Paris Collaborators: URC-CIC Paris Descartes Necker Cochin, Imagine Institute, Association Française contre les Myopathies (AFM), Paris, Marie Lannelongue Hospital
Conditions	Sickle Cell Disease (SCD), Vaso-occlusive Events
Enrollment	30 participants
Start Date	2026-09
Primary Completion	2030-03 (estimated)
Study Completion	2030-03 (estimated)
Updated on ClinicalTrials.gov	2026-08-07