Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in
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
| Condition(s) | β-thalassemia |
|---|---|
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Study type | Interventional |
| Summary | 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 |
| Who can participate | 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 anot |
| Ages | 8 Years to 16 Years |
| Sex | All |
| Lead sponsor | Shenzhen Hemogen |
| Locations | Shenzhen, Guangdong, China |
| Start date | 2020-12-01 |
| NCT ID | NCT05745532 |
| Official listing | https://clinicaltrials.gov/study/NCT05745532 |