Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia

ß-thalassemia Clinical Trial

Official title:
Safety and Efficacy of Lentiviral Vector Transduction of β-globin Genetically Modified Autologous CD34+ Hematopoietic Stem Cells in Patients With Transfusion-dependent β-thalassemia

Status Not yet recruiting

Clinical Trial Summary

This study will be intented to evaluate the safety and efficacy of lentiviral vector transduction of β-globin genetically modified autologous CD34+ hematopoietic stem cells in patients with transfusion-dependent β-thalassemia.

Clinical Trial Description

This is an open-label, single-dose study of BD211 in patients with transfusion-dependent β-thalassemia aged 14 to 35 years. It is estimated that 10 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 24 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	NCT05776173
Study type	Interventional
Source	Shanghai BDgene Co., Ltd.
Contact	Sujiang Zhang, MD
Phone	17717285030
Email	zbruce.zhang@hotmail.com
Status	Not yet recruiting
Phase	N/A
Start date	March 2023
Completion date	October 2026

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT00560820` - Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overload	Phase 1
Recruiting	`NCT05864170` - the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia	Early Phase 1
Completed	`NCT04054921` - Safety Study for Beta Thalassemia Subjects on PTG-300	Phase 2
Not yet recruiting	`NCT05773729` - Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia	N/A
Completed	`NCT03802201` - Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia	Phase 2
Recruiting	`NCT05745532` - Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients	Early Phase 1
Not yet recruiting	`NCT03358498` - Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving Deferasirox	N/A
Recruiting	`NCT05494333` - Correlation Between Pulmonary Functions and Physical Fitness in Children With β-thalassemia