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
Trial status: Open for Enrollment
Why is this study being done?
The primary hypothesis of the study is that treatment with either once-daily or twice-daily pacritinib results in a greater proportion of patients with thrombocytopenia and myelofibrosis achieving ≥ 35% reduction in spleen volume from baseline to Week 24 than treatment with Best Available Therapy, and a greater proportion of patients achieving a ≥ 50% reduction in total symptom score from baseline to Week 24 as measured by the Myeloproliferative Neoplasm Symptom Assessment Form 2.0.
Who is eligible to participate?
- 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
- 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