This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients
8-16 years old. Subject and/or subject's legal guardian fully understand and voluntarily sign informed consent;
Clinically diagnosed as transfusion-dependent β-thalassemia major;
With sufficient RBC infusion, subjects must maintain hemoglobin ≥9g/dL, serum ferritin threshold ≤ 3000 ng/mL and the liver iron overload mild or absent for at least 3 months before mobilization of hematopoietic stem cell;
Follow the arrangements for treatment and regular medical checks within two years post-transplantation
The physical condition does not meet the requirements for hematopoietic stem cell mobilization and transplantation myeloablation;
Received gene therapy and allogeneic HSCT in the past.
Have an available HLA matched donor.
Enrolling in another clinical trial.
Single group
β-globin restored autologous hematopoietic stem cells
Biological/Vaccineβ-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)
The number and the percentage of adverse events related to transplantation in 100 days will be summarized according to NCI CTCAE 5.0
Overall survival
Number of patients alive through the whole trial will be record
Proportion of engraftment
Neutrophil count \[ANC\] \>=500 /mm3 for 3 consecutive days and platelet count \[PLT\] \>20,000/mm3 for7 consecutive days
Replication competent lentivirus (RCL)
The percentage of RCL should be negative in the 24 months after transplant
The average Insertion copy number (VCN) in peripheral blood mononuclear cells
The average insertion copy number (VCN) should be ≥0.1 in peripheral blood mononuclear cells
The expression level of exogenous adult hemoglobin
Exogenous adult hemoglobin will be evaluated by globin chains and hemoglobin synthesis on peripheral blood by HPLC and the exogenous adult hemoglobin level is ≥2.0g/dL
Change from baseline in annualized frequency of packed RBC transfusions
Compare the annualized number of pRBC transfusions before gene therapy with the Month 18 and Month 24 period after transplant, the percentage change will be recorded
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