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A Phase II multi-center, open label, randomized study to assess safety and efficacy of two different schedules of oral LDE225 in adult patients with relapsed/refractory or untreated elderly patients with acute leukemia

During the last several years, substantial progress has been made regarding the biologic characterization of the acute leukemias. The traditional understanding that myeloid leukemic cells are developmentally similar to their normal hematopoietic…

A Randomized Controlled Phase 3 Study of Oral Pacritinib versus Best Available Therapy in Patients with Thrombocytopenia and Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis

The primary objective is to compare the efficacy of two dose-schedule arms(s) of pacritinib (pooled once daily [QD] and twice-daily [BID] dosing arms) with that of best available therapy (BAT) in patients with thrombocytopenia and primary…

A Randomised Feasibility n-of-1 Trial of Weekly-Interval Red Cell Transfusion in myelodysplastic syndromes; Red blood cell transfusion schedule in myelodysplastic syndromes (REDDS): study 2

This protocol describes a two-part study to assess red blood cell (RBC) transfusion schedules in patients with transfusion-dependent myelodysplastic syndromes (MDS).The first part of the study is a pilot n-of-1 study to assess the feasibility of a…