A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With b-thalassemia Treated With Base-edited Autologous Hematopoietic Stem Cell (CS-101) Transplantation.

This article provides information about a newly launched clinical trial, NCT06479616, which aims to evaluate the long-term safety and efficacy of CS-101 in participants with b-thalassemia. The trial will follow subjects who received CS-101 in a previous study, CS-101-03 (NCT06065189), for up to 2 years post-infusion. The primary outcome measures include the occurrence of new malignancies and hematologic disorders, achieving transfusion independence, frequency and severity of adverse events, and all-cause death. The study is currently recruiting participants in China. [Extracted from the article]