NCT05773729

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

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: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Shanghai BDgene Co., Ltd. · Industry
• Sex: All
• Age: 3 Years – 18 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 18 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.

Conditions

• β-thalassemia

Interventions

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

Timeline

• Start date: 2023-09-15
• Primary completion: 2026-03-01
• Completion: 2026-10-01
• First posted: 2023-03-17
• Last updated: 2024-06-14

Locations

1 site across 1 country: China