Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified
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|ClinicalTrials.gov Identifier: NCT03276455|
Recruitment Status : Not yet recruiting
First Posted : September 8, 2017
Last Update Posted : September 8, 2017
|Condition or disease||Intervention/treatment||Phase|
|Beta Thalassemia Major||Genetic: Autologous CD34+ cells genetically modified||Phase 1 Phase 2|
Beta-thalassemia major is a life-threatening genetic disease of red cell malfunction. It is caused by mutations in the beta-globin gene which encodes the beta-globin protein, leading to the ineffective erythropoiesis, hemolysis and anemia. Transplantation of allogeneic hematopoietic stem cells (HSCT) is the only available cure which is, however, has the significant risk of transplant related mortality, graft versus host disease and limited source. Therefore, transplantation of autologous hematopoietic stem cells will be an attractive therapeutic treatment for beta-thalassemia major patients. 10 patients will be treated with genetically modified autologous hematopoietic stem cells which transduced with lentiviral vector encoding for beta-globin gene.
Patients will participate for this study for 3 years.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Evaluation of Safety and Efficacy of Transplantation of Autologous Hematopoietic Stem Cell Genetically Modified in Beta-Thalassemia Major|
|Estimated Study Start Date :||September 15, 2017|
|Estimated Primary Completion Date :||September 15, 2020|
|Estimated Study Completion Date :||September 15, 2021|
Beta-thalassemia major subjects who are 8 years age or older are transplated by autologous CD34+ cells genetically modified(autologous hematopoietic stem cell transduced with lentiviral vector encoding the therapeutic beta-globin gene).
Genetic: Autologous CD34+ cells genetically modified
Autologous hematopoietic stem cell transduced with lentiviral vector encoding the therapeutic beta-globin gene. The target dose in the transduced product is 3x10^6 cells/Kg CD34+ cells, with a minimum dose of 2 x 10^6/Kg and a maximum dose of 20 x 10^6/Kg, depending on the yield of cells. The product will be injected intraosseously following intravenous BU ±Flu ±Cy.
- incidence of adverse events [ Time Frame: 0-36 months after transplantation ]Evaluate the safety of treatment with transplantation of autologous hematopoietic stem cell transduced with lentiviral vector encoding the therapeutic beta-globin gene as measured by the incidence of adverse events.
- hemoglobin conten [ Time Frame: 3-36 months after transplantation ]Evaluate the efficacy of treatment with transplantation of autologous hematopoietic stem cell transduced with lentiviral vector encoding the therapeutic beta-globin gene by the detection hemoglobin content of peripheral blood cells.
- Hematopoietic stem cell engraftment [ Time Frame: 42 days after transplantation ]Success and kinetics of hematopoietic stem cell engraftment.
- RCL [ Time Frame: 1-36 months after transplantation ]The generation of a replication-competent lentivirus (RCL).
- VCN [ Time Frame: 1-36 months after transplantation ]Quantify gene transfer efficiency and expression by evaluation of average vector copy number(VCN) in blood or bone marrow cells.
- bete-globin content [ Time Frame: 1-36 months after transplantation ]Evaluate the efficacy of treatment by detection of bete-globin content of HGB of peripheral blood cells.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03276455
|Contact: Chunfu Li, PhDemail@example.com|
|Contact: Zhiyong Pengfirstname.lastname@example.org|
|Guangzhou, Guangdong, China, 510515|
|Contact: ChunFu Li, PhD 86-020-61641921 email@example.com|
|Principal Investigator: ChunFu Li, PhD|