Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia
Trial Parameters
Brief Summary
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.
Eligibility Criteria
Inclusion Criteria: 1. Ages 3 to 18 years old, including: The parents or legal guardians must be able to understand and provide ICFs. If available, it is strongly recommended that children aged ≥8 years in treatment decisions and obtain written ICFs and be clearly documented; Diagnosed as Transfusion Dependent β-thalassemia with any genotype (β0, β+, βE/β0, βS/S, βS/β0, βS/β+), confirmed the Hb analysis. No alfa chain genetic abnormalities. Subjects must stabilize and maintain an appropriate iron chelation regimen. Transfusion-dependent types are defined as requiring at least 100 mL/kg/ year of red blood cells (pRBCs). 2. No eligiblity for allogeneic hematopoietic stem cell transplantation. 3. The treatment of erythrocyte maturation agent luspatercept cannot be financially supported. 4. The subjects' parents/legal guardians must be willing and able to follow the study procedures in the study protocol. 5. Good organs' functions. 6. Having complete medical records including a history of