Art

Reichweite

Status

Start

Ende

Finanzierungsart

Studiendesign

Schlagwörter (Tags)

Kurzbeschreibung/Zielsetzung

Myelofibrosis (MF) is defined by progressive bone marrow (BM) fibrosis and a consecutive reduction of blood cells. The disrup-tion of the medullary erythropoietic niche is the primary mecha-nism governing the bone marrow failure and anemia, which typify MF. Nearly 40% of MF patients have hemoglobin (Hb) levels < 10 g/dL at diagnosis. Furthermore, anemia is the disease feature most consistently associated with poor prognosis in MF. Ruxolitinib demonstrates improvements in splenomegaly and constitutional symptoms, however, does not improve anemia.
The purpose of this study is to investigate the safety, pharmacokinetics (PK) and preliminary efficacy of combinations treatment of ruxolitinib with 3 novel compounds: siremadlin, crizanlizumab and MBG453 in MF subjects. These combination therapies may deliver transformational clinical benefits such as improvement of progression free survival (PFS) as a consequence of superior disease control or reduction of the malignant clone, associated with an improvement of cytopenia and in particular anemia, as well as improvement in quality of life (QoL) as captured by relevant patient reported outcomes measurements (PROs)