This study aims to determine if treatment with a single infusion of autologous CD34+ HSC cells transduced with the lentiviral vector containing shmiR targeting BCL11A will lead to a complete absence of severe vaso-occlusive events (VOEs) in the period from Month 6 to Month 24 after receiving the gene therapy in participants with sickle cell disease, with genotype HbSS or HbS/B0 thalassemia.
Study treatment includes mobilization and collection of CD34+ HSPCs, myeloablative conditioning with busulfan administered on days -5 to -2 prior to infusion of gene therapy, and one 30-45 minute infusion after standard pre-hydration and premedication. After infusion of transduced cells, red cell transfusions will be utilized to maintain healthy and stable hemoglobin levels.
Sponsor
National Marrow Donor Program
Status of enrollment
Accepting new patients
Ages Eligible for Study
For people ages 13-40
Disease indication
Sickle Cell Anemia
Principal Investigator
Mark Walters, MD
Contact
Marci Moriarty, RN, BSN: [email protected]
Additional study eligibility details can be found at UCSF Clinical Trials.