Long-Term Safety and Effectiveness Study of Betibeglogene Autotemcel Treatment in Beta-Thalassemia Patients

What this Trial Is About

Researchers are collecting long-term real-world data on people with beta-thalassemia who have received betibeglogene autotemcel beti-cel treatment after it became available on the market. The study aims to assess the safety of beti-cel over a period of up to 15 years, including monitoring for any new cancers, as well as evaluating how effective the treatment is over time. Participants who have been treated with beti-cel at participating centers in the US will be followed without any additional interventions. This observational registry study tracks patients for up to 15 years after their beti-cel infusion, gathering health information to understand long-term outcomes. During the study, researchers will collect data on any adverse events, serious health issues, and how many participants achieve independence from blood transfusions. All participants will be monitored regularly through their usual medical care with a hematologist in the US. The study focuses on safety and effectiveness outcomes over the long term, with no exclusion criteria for participation.

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel