Phase 1 Study of BD211 Gene Therapy via Intravenous Infusion for Treating Transfusion-Dependent Beta-Thalassemia in Patients Aged 3 to 35

What this Trial Is About

Researchers are evaluating the safety, tolerability, and effectiveness of a gene therapy treatment called BD211 for patients aged 3 to 35 years with transfusion-dependent beta-thalassemia. This condition requires regular blood transfusions, and the study aims to assess how well the gene-modified stem cells work and how safely they can be used. The study is a Phase 1, open-label trial sponsored by Shanghai BDgene Co., Ltd., focusing on patients who need frequent transfusions and have limited treatment options. The treatment involves a single intravenous infusion of BD211, which consists of autologous CD34 hematopoietic stem cells that have been genetically modified with a lentiviral vector to produce healthy beta-globin. Participants receive one dose of at least 5 million cells per kilogram of body weight. The study includes an 18-month follow-up period to monitor safety endpoints and effectiveness, including the ability to reduce or eliminate the need for transfusions. During the study, participants undergo regular assessments to track neutrophil and platelet engraftment, transfusion independence, blood hemoglobin levels, ferritin levels, and the presence of the beta-globin protein in blood. Researchers also monitor for adverse events, survival rates, hospitalizations, and any signs of complications related to the gene therapy. Participants are expected to comply with scheduled visits, laboratory tests, and long-term follow-up assessments to help determine the treatments overall safety and efficacy.

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