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 Disease Splenomegaly Pacritinib MPN-SAF MPN-SAF TSS Anemia Myeloproliferative Neoplasm Spleen volume Thrombocytopenia SB1518
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 327
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 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 primary or secondary myelofibrosis. Approximately 322 eligible patients will be randomized in a 2:1 allocation to pacritinib (400mg QD) or BAT (includes any physician-selected treatment for myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases)). 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: BAT includes any physician-selected treatment for primary or secondary myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases)

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

Protocol document: