Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified
Last updated on August 2021Recruitment
- Recruitment Status
- Not yet recruiting
Summary
- Conditions
- Recurrent Adult Soft Tissue Sarcoma
- Stage III Adult Soft Tissue Sarcoma
- Stage IV Adult Soft Tissue Sarcoma
- Beta Thalassemia Major
- Type
- Interventional
- Phase
- Phase 1Phase 2
- Design
- Allocation: N/A
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
Participation Requirements
- Age
- Between 8 years and 125 years
- Gender
- Both males and females
Description
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...
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.
Tracking Information
- NCT #
- NCT03276455
- Collaborators
- National Comprehensive Cancer Network
- Guangdong Yike Gene Science and Technology CO.,Ltd
- Investigators
- Principal Investigator: Warren Chow City of Hope Comprehensive Cancer Center
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