An Open-label Clinical Trial of Gene Therapy (Vebeglogene Autotemcel) Using Beta-globin Lentiviral Vector for Treatment of Transfusion-Dependent Beta-Thalassemia

What this Trial Is About

Researchers are evaluating the safety and effectiveness of a gene therapy product called Vebeglogene Autotemcel for patients with transfusion-dependent beta-thalassemia, a blood disorder requiring regular red blood cell transfusions. This open-label interventional study focuses on using the patient's own hematopoietic stem and progenitor cells (HSPCs) that are genetically modified to carry a functional hemoglobin gene (HBB). The study is led by Lantu Biopharma and aims to assess both safety and treatment outcomes over time. The treatment involves collecting the participant's HSPCs and modifying them ex vivo with a lentiviral vector carrying the functional HBB gene. After a myeloablative conditioning process lasting about 10 days, participants receive a one-time infusion of the modified cells at a dose of at least 5 million cells per kilogram of body weight. The study timeline includes approximately 30 days for screening and baseline assessments, around 60 days for mobilization and manufacturing of the therapy product, followed by the conditioning, infusion, and a 24-month observation period to monitor effects and safety. Participants will undergo various assessments during the study, including monitoring hemoglobin levels to see how long they maintain at least 9.0 g/dL without needing transfusions, tracking adverse events, and measuring transfusion needs compared to prior records. Blood tests will evaluate engraftment success and vector presence over the 24-month period. The total study duration for each participant is about 27 months, ensuring thorough safety and efficacy evaluation of the gene therapy treatment.

An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia