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
Eligibility
Sex
ALL
Min age
8 Years
Max age
16 Years
Healthy volunteers
No
Inclusion Criteria:
* 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
Exclusion Criteria:
* 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.
* Other unsuitable conditions identified by doctors.
Primary outcome measure(s)
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) — 0-100 days 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 — 0-24 months Number of patients alive through the whole trial will be record
Proportion of engraftment — 0-24 months Neutrophil count \[ANC\] \>=500 /mm3 for 3 consecutive days and platelet count \[PLT\] \>20,000/mm3 for7 consecutive days
Replication competent lentivirus (RCL) — 0-24 months The percentage of RCL should be negative in the 24 months after transplant
Dynamics of viral integration sites (VIS) — 0-24 months Evaluation of the percentage of participants without abnormal clonal proliferation and polyclonal engraftment . More than 1000 VIS retrieved from peripheral blood should be checked.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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