Study Comparing Luspatercept with Low-Dose Thalidomide Versus Luspatercept Alone in Adults with Transfusion-Dependent Beta-Thalassemia

What this Trial Is About

This research aims to evaluate the clinical efficacy and safety of combining luspatercept with low-dose thalidomide compared to using luspatercept alone in adults with transfusion-dependent b2-thalassemia major. This inherited blood disorder requires regular red blood cell transfusions and has limited treatment options, making this study important for improving patient care. The trial is a prospective, multicenter, randomized, double-blind, placebo-controlled phase 2 study. Participants are randomly assigned in a 11 ratio to either receive luspatercept combined with low-dose thalidomide or luspatercept with a placebo. Luspatercept is given at a starting dose of 1.0 mgkg every 21 days, while thalidomide or placebo is given daily at 50 mg. Both treatments last for 48 weeks, and participants may continue best supportive care like transfusions, iron chelation, and other therapies as needed. The study includes a screening period, treatment phase, and follow-up at weeks 12, 24, 36, and 48. During the study, participants undergo physical exams, vital sign checks, blood tests, transfusion tracking, iron level monitoring, quality-of-life assessments, and safety evaluations. Researchers measure how well the treatment reduces the need for blood transfusions, with the primary outcome focusing on transfusion reduction between weeks 13 and 24. Secondary outcomes assess longer-term transfusion reduction, iron metabolism, hemolysis, and adverse events. Data is collected throughout the treatment and follow-up periods for detailed analysis.

Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients