Study of Luspatercept Treatment for Adults With Clonal Cytopenias of Undetermined Significance CCUS

What this Trial Is About

Researchers are evaluating how well the drug luspatercept improves low blood cell counts in people with clonal cytopenias of uncertain significance CCUS. This phase 2 clinical trial aims to answer key questions about the number of patients who experience blood count improvements within 24 weeks, the duration of these improvements, and the time until disease progression to more severe conditions such as myeloid disorders. The study also monitors changes in hemoglobin levels, transfusion needs, quality of life, and potential side effects including progression to leukemia or heart-related events. Participants will receive luspatercept injections at a dose of 1 mgkg intravenously every three weeks. Treatment and monitoring visits also occur every three weeks. The study tracks responses at multiple time points including 12, 24, and 48 weeks, with assessments of blood counts, transfusion dependence, and quality of life scores. Safety is closely monitored throughout the treatment period. During the study, participants will visit the clinic every three weeks for their injections and evaluations. Researchers will perform blood tests to measure various blood cell levels, hemoglobin, and cardiac biomarkers. Participants will complete questionnaires to assess their well-being and energy levels. The main outcome is the number of patients achieving hematologic improvement by 24 weeks, with additional measures of response duration and safety events. The study is expected to continue until February 2028.

Luspatercept for Clonal Cytopenias of Uncertain Significance