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.
No eligiblity for allogeneic hematopoietic stem cell transplantation.
The treatment of erythrocyte maturation agent luspatercept cannot be financially supported.
The subjects' parents/legal guardians must be willing and able to follow the study procedures in the study protocol.
Good organs' functions.
Availability of voluntary, fully HLA-matched hematopoietic cell donors, unless recommended for inclusion by the Monitoring Committee.
HIV-1 and HIV-2 were positive, and / or HTLV-1, HTLV-2 and VSV-G antibodies were positive.
An active bacterial, viral, fungal or parasitic infection.
Contraindicated for the extraction of bone marrow under anesthesia.
Single group
BD211
GeneticGenetically modified CD34+ autologous stem cells were transfused intravenously with single dosing.
Percentage of treated participants with Transfusion-Dependent β-Thalassemia (TDT) who achieved transfusion independence (TI) for at least 6 months
TI defined as peripheral blood weighted average hemoglobin (Hb) \> or = 9 g/dL without packed red blood cell (pRBC) transfusion for 60 days after BD211 treatment, and transfusion is continuously halted for 12 months.
Hb (g/dL) level between 12 and 24 months after BD211 treatment compared with baseline Hb level
Hb (g/dL) in 12, 15, 18, 21 and 24 months after drug product infusion are tested and calculated.
Parameters of efficacy related to TI achievement after BD211 treatment
including a. Duration to achieve TI; b. Time from BD211 infusion to last transfusion; c. Time to reach TI from BD211 infusion; d. Mean weighted Hb (g/dL) during TI; and e. Proportion of patients achieving TI at 24 month after BD211 treatment.
Parameters of efficacy related to reduced blood transfusion after BD211 treatment
Including a. Using the transfusion volume from the 2 years prior to the participant's enrollment as a baseline, compared with the transfusion volume between 12 and 24 months after BD211 treatment (mL/kg/month, mL/kg/year), the proportion of participants categorized based on a reduction in transfusion volume of 50%, 75%, 90%, and 100%; b. Using the number of transfusions from the 2 years prior to the participant's enrollment as a baseline, comparing the changes in the volume of pRBC transfusions at 12 and 24 months after BD211 treatment; c. Using the transfusion requirement volume from the 2 years prior to the participant's enrollment as a baseline, comparing the changes in the pRBC transfusion requirement volume at 12 and 24 months after BD211 treatment.
Parameters of iron overload after BD211 treatment
Including a. Time from the last iron-chelation treatment to the last follow-up within 24 months; b. Decrease in serum ferritin level compared from baseline to 6 months and 12 months after BD211 infusion; c. Number and proportion of participants who did not require iron-chelation treatment for at least 6 months after BD211 infusion; d. Comparison of cardiac MRI T2 values at the end of the 24 month after BD211 treatment with the baseline values.
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Shanghai BDgene Co., Ltd.
Lead sponsor
Shanghai Children's Medical Center
Collaborator
