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.
The tumor genes chip detection results about acute leukemia and myeloid tumor gene mutations (panel) showed no abnormality.
There were candidates for HLA gene semi-compatible hematopoietic stem cell transplantation.
No eligiblity for allogeneic hematopoietic stem cell transplantation.
The treatment of erythrocyte maturation agent luspatercept cannot be financially supported.
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.
Red blood cell (RBCs) transfusion requirements, whether reaching TI
Comparison of blood volume and number of transfusions in the 2 years prior to participants enrolment as baseline with blood volume and number of transfusions up to months 6, 12, and 24 after receiving transfusion of the BD211. TI defined as peripheral blood weighted average hemoglobin (Hb) \> or = 9 g/dL without pRBCs transfusion for 60 days after BD211 treatment, and transfusion is continuously halted for 12 months.
Total hospitalizing days at 6, 12, and 24 months (discharge after transplant)
Total hospitalizing days at 6, 12, and 24 months after transplantation were counted.
Percentage of treated participants with Transfusion-Dependent β-Thalassemia (TDT) who achieved transfusion independence for at least 6 months
TI defined as peripheral blood weighted average hemoglobin (Hb) \> or = 9 g/dL without pRBCs transfusion for 60 days after BD211 treatment, and transfusion is continuously halted for 12 months.
Change in RBCs infusion from baseline at 6 to 24 months
The quantity of RBCs transfusions at 6 to 24 months will be measured and compared with baseline level.
Mean Hb (g/dL) at 6, 12 and 24 months after treatment
Mean Hb (g/dL) in 6, 12, and 24 months after drug product infusion are tested and calculated.
Change in ferritin/liver iron levels from baseline
The Ferritin/liver iron levels are assayed per protocol plan, and evaluate the changes from those baseline level.
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Shanghai BDgene Co., Ltd.
Lead sponsor
Ruijin Hospital
Collaborator
