Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia
Condition: Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
MeSH terms: Polycythemia Vera; Thrombocytopenia; Primary Myelofibrosis; Polycythemia; Thrombocytosis; Thrombocythemia, Essential
Intervention: Pacritinib; Best Available Therapy
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 311.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
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

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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Intermediate -1 or -2 or high-risk Myelofibrosis (per Passamonti et al 2010)

          -  Thrombocytopenia (platelet count ≤ 100,000/µL) at any time after signing informed
             consent

          -  Palpable splenomegaly ≥ 5 cm on physical examination

          -  Total Symptom Score ≥ 13 on the MPN-SAF TSS 2.0, not including the inactivity question

          -  Patients who are platelet or red blood cell transfusion-dependent are eligible

          -  Adequate white blood cell counts (with low blast counts), liver function, and renal
             function

          -  At least 6 months from prior splenic irradiation

          -  At least 1-4 weeks since prior myelofibrosis therapy, including any erythropoietic or
             thrombopoietic agent

          -  Not pregnant, not lactating, and agree to use effective birth control

          -  Able and willing to undergo frequent MRI or CT assessments and complete symptom
             assessments using a patient-reported outcome instrument

        Exclusion Criteria:

          -  Prior treatment with more than 2 JAK2 inhibitors or with pacritinib

          -  There is no maximum cumulative prior JAK2 inhibitor treatment

          -  History of (or plans to undergo) spleen removal surgery or allogeneic stem cell
             transplant

          -  Ongoing gastrointestinal medical condition such as Crohn's disease, Inflammatory bowel
             disease, chronic diarrhea, or constipation

          -  Active bleeding that requires hospitalization during the screening period

          -  Cardiovascular disease, including recent history or currently clinically symptomatic
             and uncontrolled: congestive heart failure, arrhythmia, angina, QTc prolongation or
             other QTc risk factors, myocardial infarction

          -  Other malignancy within last 3 years other than certain limited skin, cervical,
             prostate, breast, or bladder cancers

          -  Other ongoing, uncontrolled illnesses (including HIV infection and active hepatitis A,
             B, or C), psychiatric disorder, or social situation that would prevent good care on
             this study

          -  Life expectancy < 6 months