NCT06465550

A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major

A Phase 1 Clinical Trail of the Safety and Efficacy of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Intravenous Infusion for the Treatment of Transfusion-dependent β-thalassaemia Patients

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Shanghai BDgene Co., Ltd. · Industry
• Sex: All
• Age: 3 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• β-thalassemia

Interventions

Type	Name	Description
GENETIC	BD211	Genetically modified CD34+ autologous stem cells were transfused intravenously with single dosing.

Timeline

• Start date: 2024-01-05
• Primary completion: 2026-12-01
• Completion: 2026-12-01
• First posted: 2024-06-20
• Last updated: 2024-06-24

Locations

3 sites across 1 country: China