Brief Summary
This study (study ID PAC203 North America; PAC303 ex-North America) is evaluating 200 mg BID of pacritinib compared to physician’s choice (P/C) therapy in patients with MF and severe thrombocytopenialow levels of platelets in the blood (platelet count <50,000/μL). Approximately 399 patients in total will be enrolled, randomized 2:1 to either pacritinib (approximately 266 patients) or to P/C therapy (approximately 133 patients)
Condition or disease: Primary Myelofibrosis/Post-Polycythemia Vera Myelofibrosis/ Post-essential Thrombocythemia Myelofibrosis
Intervention/treatment: Drug-Pacritinib
Intervention / Treatment
- Drug: Pacritinib
- Drug: Physician’s Choice medications
Inclusion Criteria
- Primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF (as defined by Tefferi and Vardiman 2008
- Platelet count of <50,000/μL at Screeningtesting for cancer or conditions that can lead to cancer before symptoms appear, also known as cancer screening (Day -35 to Day -3)
- Dynamic International Prognostic Scoring System Intermediate-1, Intermediate-2, or High-Risk (Passamonti et al 2010
- Palpable splenomegalyenlargement of the spleen ≥5 cm below the lower costal margin (LCM) in the midclavicular line as assessed by physical examinationan examination of your current symptoms, affected area(s) and overall medical history
- TSS of ≥10 on the MPN-SAF TSS 2.0 or a single symptom score of ≥5 or two symptoms of ≥3, including only the symptoms of left upper quadrant pain, bone pain, itching, or night sweats.The TSS criteria need only to be met on a single day.
- Age ≥18 years
- Eastern Cooperative Oncologythe study, diagnosis and treatment of cancer Group performance status 0 to 2
- Peripheral blast count of <10% throughout the Screening period prior to randomization
- Absolute neutrophil count of ≥500/µL
- Left ventricular cardiac ejection fraction of ≥50% by echocardiograma type of ultrasound that uses sound waves to create detailed images of the heart to assess heart structure, function and blood flow or multigated acquisition scan
- Adequate liver and renal function, defined by liver transaminases (aspartate aminotransferase [AST]/serum glutamic-oxaloacetic transaminase [SGOT] and alanine aminotransferase [ALT]/serum glutamic pyruvic transaminase [SGPT]) ≤3 × the upper limit of normal (ULN) (AST/ALT ≤5 × ULN if transaminase elevation is related to MF), total bilirubin ≤4 x ULN (in cases where total bilirubin is elevated, direct bilirubin ≤4 × ULN, is required) and creatinine ≤2.5 mg/dL
- Adequate coagulation defined by prothrombin time/international normalized ratio and partial thromboplastin time ≤1.5 × ULN
- If fertile, willing to use highly effective birth control methods during the study
- Willing to undergo and able to tolerate frequent MRI or CT scan assessments during the study
- Able to understand and willing to complete symptom assessments using a patient-reported outcome instrument
- Provision of signed informed consenta process in which a patient receives detailed information about a procedure or treatment, including its potential risks, benefits, and alternatives. The patient then has the opportunity to understand the information and voluntarily agree to the procedure or treatment.