Conditions: Primary Myelofibrosis Post-polycythemia Vera Myelofibrosis Post-essential Thrombocythemia Myelofibrosis
Keywords: Myelofibrosis Post-Polycythemia Vera Myelofibrosis Post-Essential Thrombocythemia Myelofibrosis Primary Myelofibrosis Polycythemia Vera Essential Thrombocythemia Bone Marrow Disease Hematologic Diseases Hemorrhagic Disorders Splenomegaly Pacritinib MPN-SAF MPN-SAF TSS Anemia Myeloproliferative Neoplasm Spleen volume Thrombocytopenia SB1518
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 311
Healthy volunteers: no

Brief summary:
Phase 3, randomized, controlled study to evaluate the safety and efficacy of oral pacritinib compared to Best Available Therapy (BAT) in patients with thrombocytopenia and primary or secondary myelofibrosis.

Detailed description:
Multicenter, randomized, controlled, phase 3 trial comparing the safety and efficacy of pacritinib with that of BAT in patients with thrombocytopenia and primary or secondary myelofibrosis. Approximately 300 eligible patients will be randomized in a 1:1:1 allocation to pacritinib 400 mg dosed QD, pacritinib 200 mg dosed BID, or BAT (includes any physician-selected treatment for myelofibrosis, such as approved JAK2 inhibitors administered according to package insert for patients with thrombocytopenia, and may include any treatment received before study entry). Spleen volume will be measured by MRI or CT at baseline and every 12 weeks thereafter. An independent radiology facility (IRF), blind to treatment assignments, will measure spleen volumes. Patients will also be followed for safety, Leukemia Free Survival (LFS), Overall Survival (OS), frequency of red blood cell (RBC) and platelet transfusions, and other exploratory endpoints. An Independent Data Monitoring Committee (IDMC) will evaluate the safety of pacritinib.

Arms:
arm 1: Pacritinib 400 mg QD arm 2: Pacritinib 200 mg BID arm 3: BAT includes any physician-selected treatment for myelofibrosis, such as approved JAK2 inhibitors administered according to package insert for patients with thrombocytopenia, and may include any treatment received before study entry.

Interventions: intervention 1: Pacritinib intervention 2: Best Available Therapy
Intervention descriptions: intervention 1: None intervention 2: None

Protocol document: