An open-label, randomized, Phase 2, parallel, dose-ranging, multicenter, study of sotatercept for the treatment of patients with anemia and low- or intermediate-1 risk myelodysplastic syndromes or non-proliferative chronic myelomonocytic leukemia (CMML)
The primary objective of this study is to determine a safe, tolerable and effective dose of sotatercept that results in the greatest frequency of erythroid hematological improvement.
This study is for patients with the diagnosis of MDS or non-proliferative CMML that meet the International Prognostic Scoring System criteria for low or intermediate-1 risk disease. Subjects must have anemia that requires transfusions. This is defined as at least 2 units of RBCs within 84 days of enrollment for a hemoglobin less than or equal to 9 g/dL. Subjects must also have no response to treatment with epoetin alpha, no response to darbopoetin alpha, or have a low chance of response to erythroid stimulating factors reflected by EPO concentration of greater than 500 mU/ml. Patients with MDS with chromosome 5q minus are not eligible. Patients much have a platelet count greater than 30,00/uL. Patients with uncontrolled hypertension are not eligible.
There are 2 parts to this study. Part 1: Patients will be stratified into 3 treatment groups. The treatment groups are 0.1 mg/kg sotatercept, 0.3 mg/kg sotatercept, and 0.5 mg/kg sotatercept. Patients get this study drug delivered subcutaneously every 3 weeks for 5 cycles. If a subject is determined to be having a response (erythroid hematological improvement) after 5 cycles will continue onto an extension study and continue to receive sotatercept until no longer beneficial. Part 2: The treatment group from part 1 that is determined safe and has the most HI-E will be the dose used in part 2. Patients will be administered sotatercept every 3 weeks subcutaneously for 5 cycles. If a subject is determined to be having a response (erythroid hematological improvement) after 5 cycles will continue onto an extension study and continue to receive sotatercept until no longer beneficial.
04/26/2015 04:01 AM