Beta Thalassemia Major Clinical Trial
Official title:
Evaluation of Safety and Efficacy of Transplantation of Autologous Hematopoietic Stem Cell Genetically Modified in Beta-Thalassemia Major
This is a single group, open label study in 10 subjects who are 8 years of age or older with beta-thalassemia major. The objective of this study is to evaluate the safety and efficacy of autologous hematopoietic stem cell transduced with lentiviral vector for the treatment of beta-thalassemia major.
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.
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