NCT03351829

Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector

• Status: Unknown
• Phase: N/A
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Shenzhen Geno-Immune Medical Institute · Academic / Other
• Sex: All
• Age: 4 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.

Detailed description

Thalassemia is considered the most common genetic disorder worldwide. Beta-thalassemia is caused by mutations in the beta-globin gene which encodes the beta-globin protein, leading to the ineffective erythropoiesis, hemolysis and anemia. Currently, the only cure for thalassemia is bone marrow transplantation from a related, compatible donor, which has, however, the significant risk of transplant related mortality, graft versus host disease and limited source. Therefore, gene transfer, achieved by transplantation of the patient's own stem cells that have been genetically-modified with the corrected gene, could potentially cure thalassemia. This study will use an experimental gene transfer procedure performed in a laboratory to insert the related gene into the participant's autologous stem cells using a self-inactivating lentiviral vector. The purpose of this study is to evaluate the safety and effectiveness of the gene transfer procedure and to determine the ability of the gene-corrected cells at generating new, healthy blood cells in individuals.

Conditions

• Beta-Thalassemia

Interventions

Type	Name	Description
GENETIC	Gene-modified autologous stem cells	1 infusion for 5x10\^6\~1x10\^7 gene-modified cells; or more infusions depending on the circumstances

Timeline

• Start date: 2017-12-01
• Primary completion: 2019-01-01
• Completion: 2020-12-31
• First posted: 2017-11-24
• Last updated: 2017-11-30

Locations

1 site across 1 country: China