Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-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 6 to 35 years old, including: Subjects should be able to provide an ICF. 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. The tumor genes chip detection results about acute leukemia and myeloid tumor gene mutations (panel) showed no abnormality. 3. There were candidates for HLA gene semi-compatible hematopoietic stem cell transplantation. 4. No eligiblity for allogeneic hematopoietic stem cell transplantation. 5. The treatment of erythrocyte maturation agent luspatercept cannot be financially supported. 6. The investigator confirmed that subject was willing to follow the research procedures. 7. Having complete medical records including a history of blood trans