Oral Pacritinib Versus Best Available Therapy to Treat Myelofibrosis With Thrombocytopenia (PAC326)
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
Cell Therapeutics Inc.
300 participants
Jul 4, 2014
Interventional
Conditions
Summary
This study will compare the chemotherapy drug, Pacritinib, against the best available treatment for Thrombocytopenia and Myelofibrosis. You may be eligible to join this study if you aged 18 years or above and have been diagnosed with thrombocytopenia and/or myelofibrosis. Participants in this study are randomly allocated (by chance) to one of three groups. Participants in the first group will receive one dose of 400mg of oral Pacritinib for 24 weeks or until progression has occurred. Participants in the second group will receive two doses of 200mg of oral Pacritinib daily for up to 24 weeks or until progression occurs. Participants in the third group will receive best available treatment - which will be chosen by your treating physician. Participants will undergo magnetic resonance imaging (MRI) or computed tomography (CT) scans to determine spleen volume and will be assessed for total symptom score using the Myeloproliferative Neoplasm Symptom Assessment Form 2.0. These assessments will occur 12 weekly. Patients may crossover from the BAT (Best available treatment) arm to Pacritinib at the discretion of the Investigator.
Eligibility
Inclusion Criteria10
- Intermediate -1 or -2 or high-risk Myelofibrosis (per Passamonti et al 2010)
- Thrombocytopenia (platelet count greater than 100,000/microlitre) at any time after signing informed consent
- Palpable splenomegaly greater than 5 cm on physical examination
- Total Symptom Score less than 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 Criteria9
- Prior treatment with more than 2 JAK2 inhibitors or with pacritinib
- More than 6 months of 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
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Interventions
Pacritinib (oral) 400mg QD or BID daily for 24 weeks, PK (Pharmocokinetic)/PD (Pharmacodynamic) analysis and drug tablet return
Locations(6)
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ACTRN12614000740695