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Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified

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

Clinical Trial Summary

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.

Clinical Trial 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) 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 Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03276455
Study type Interventional
Source Nanfang Hospital of Southern Medical University
Contact Chunfu Li, PhD
Phone 86-020-61641921
Email chunfugzcn@126.com
Status Not yet recruiting
Phase Phase 1/Phase 2
Start date September 15, 2017
Completion date September 15, 2021
View Details
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