ß-Thalassemia Major Clinical Trial
Official title:
A Single Center, Open Label Study to Evaluate the Safety and Efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients
Verified date | September 2020 |
Source | BGI-research |
Contact | Jing Li, PhD |
Phone | 13510560664 |
lijing4[@]genomics.cn | |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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
Status | Not yet recruiting |
Enrollment | 10 |
Est. completion date | November 30, 2024 |
Est. primary completion date | November 30, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 8 Years to 16 Years |
Eligibility | 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 ß-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. |
Country | Name | City | State |
---|---|---|---|
China | Beijing Genomics Institute At Shenzhen | Shenzhen | Guangdong |
Lead Sponsor | Collaborator |
---|---|
BGI-research | Shenzhen Children's Hospital |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | 0-100 days | |
Primary | Overall survival | Number of patients alive through the whole trial will be record. | 0-24 months | |
Primary | Proportion of engraftments | Neutrophil count [ANC] >=500 /mm3 for 3 consecutive days and platelet count [PLT] >20,000/mm3 for7 consecutive days. | 0-24 months | |
Primary | Replication competent lentivirus (RCL) | The percentage of RCL should be negative in the 24 months after transplant. | 0-24 months | |
Primary | Dynamics of viral integration sites (VIS) | Evaluation of the percentage of participants without abnormal clonal proliferation and polyclonal engraftment at 6, 12, 18 and 24 months after transplant. More than 1000 VIS retrieved from peripheral blood should be checked. | 0-24 months | |
Secondary | 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. | 18-24 Months | |
Secondary | 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. | 18-24 Months | |
Secondary | Change from baseline in annualized frequency and volume of packed RBC transfusions | Compare the annualized number of pRBC transfusions before gene therapy with the Month 6 and Month 24 period after transplant, the percentage change will be recorded. | 18-24 Months |
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