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A Phase 2, Multicenter, Open-Label Study To Evaluate The Efficacy And Safety Of Luspatercept (Ace-536) In Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis And Anemia With And Without Red Blood Cell-Transfusion Dependence
The main purpose of this study is to see if people with Myeloproliferative Neoplasm (MPN) Associated myelofibrosis that may or may not need blood transfusions will experience an increase of their hemoglobin or stop needing blood transfusions by taking luspatercept. The safety of luspatercept will be also evaluated in this study.
Primary Objective: The primary objective of the study is to evaluate the efficacy and safety of luspatercept for the treatment of anemia in subjects with myeloproliferative neoplasm (MPN)-associated myelofibrosis with and without red blood cell (RBC)-transfusion dependence. Secondary Objectives: To evaluate the safety of luspatercept in MPN-associated myelofibrosis. To evaluate the effect of luspatercept in MPN-associated myelofibrosis: - on the time to and duration of anemia response - on frequency of RBC transfusions and transfusion dependence - on symptom response improvement via the Myelofibrosis Symptom Assessment Form (MF-SAF) v4 - on health-related quality of life (HRQoL) via the EQ-5D-5L and Functional Assessment of Cancer Therapy - Anemia (FACT-An) questionnaires. To evaluate population pharmacokinetics of luspatercept in subjects with MPNassociated myelofibrosis with and without RBC-transfusion dependence. Explanatory Objectives: To explore the relationship between luspatercept exposure and measures of efficacy and toxicity. To explore biomarkers related to efficacy parameters and markers related to mechanism of action of luspatercept. - Activin receptor IIB ligands such as growth differentiation factor 11 (GDF11) and, - Activin B, - Inflammatory cytokines such as interleukin (IL)-6, IL-8, and tumor necrosis factor-alpha (TNF-a), - Serum markers related to fibrogenesis such as transforming growth factor-beta 1 (TGF-b1) and thrombopoietin (TPO), - Circulating blasts (CD34+ cells), - Gene expression profiling or other similar evaluation related to drug mechanism of action. To explore MPN-associated myelofibrosis-related mutations relative to disease progression and/or drug response.