Clinical Trials

Date: 2017-06-01

Type of information: Completion of patient enrollment

phase: 3

Announcement: completion of patient enrollment

Company: Celgene (USA - NJ) Acceleron Pharma (USA - CA)

Product: luspatercept

Action mechanism:

fusion protein. Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-Beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Luspatercept is currently in phase 2 clinical trials in patients with beta-thalassemia and in patients with myelodysplastic syndromes.
Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene .

Disease: beta-thalassemia

Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases

Country: Australia, Bulgaria, Canada, France, Greece, Israel, Italy, Lebanon, Malaysia, Taiwan, Thailand, Tunisia, Turkey, UK, USA

Trial details:

The BELIEVE Phase 3 trial is a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of luspatercept in patients with transfusion dependent beta-thalassemia. The primary endpoint of the study is the proportion of patients achieving a ? 33% reduction in red blood cell transfusion burden from Week 13 to Week 24 compared to the baseline 12-week period prior to treatment. Secondary endpoints include reductions in RBC transfusion burden from Week 37 to Week 48 compared to baseline. Beta-thalassemia patients in the trial had a baseline RBC transfusion burden of 6 to 20 units over the 24-week period prior to treatment. Patients were randomized 2:1, luspatercept to placebo treatment, administered subcutaneously every 3 weeks for 48 weeks. The BELIEVE study is being conducted at 73 investigational sites in 15 countries. (NCT02604433)

Latest news:

• On June 1, 2017, Celgene and Acceleron Pharma announced that they have completed target enrollment in the BELIEVE Phase 3 study of luspatercept in patients with beta-thalassemia. The Companies expect to report top-line results from the clinical trial in the middle of 2018.