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A Phase 1 Study of the Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cell in β-thalassemia Major

ß-thalassemia Clinical Trial

Official title:
A Phase 1 Clinical Trail of the Safety and Efficacy of BD211 Intravenous Infusion for the Treatment of Transfusion-dependent β-thalassaemia Patients

Clinical Trial Summary

This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.

Clinical Trial Description

This is an open-label, single-dose study of BD211 in patients with transfusion-dependent β-thalassemia aged 3 to 35 years. It is estimated that 9 subjects will be enrolled. BD211 is a gene modified gene therapy product designed to produce healthy β-globin in red blood cells in beta-thalassemia patients. The total follow-up duration was 18 months, the safe endpoints and effectiveness endpoints will be used to assess the safety and efficacy profiles in patients with transfusion-dependent β-thalassemia. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06465550
Study type Interventional
Source Shanghai BDgene Co., Ltd.
Contact fujun Li
Phone +86-19121311061
Email fujun.li@bdgene.cn
Status Recruiting
Phase Phase 1
Start date January 5, 2024
Completion date December 2026
View Details
See also
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Recruiting NCT05494333 - Correlation Between Pulmonary Functions and Physical Fitness in Children With β-thalassemia