RecruitingPhase 3ACTRN12614000740695

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

Sponsor

Cell Therapeutics Inc.

Enrollment

300 participants

Start Date

Jul 4, 2014

Study Type

Interventional

Conditions

Primary Myelofibrosis

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

Sex: Both males and femalesMin Age: 18 Yearss

Plain Language Summary

Simplified for easier understanding

Myelofibrosis is a type of bone marrow cancer that causes scar tissue to build up in the bone marrow, leading to severe fatigue, enlarged spleen, and low blood cell counts. Thrombocytopenia (low platelets) makes treatment more challenging. This study is comparing pacritinib — a newer targeted cancer drug — in two dosing schedules versus the best available standard treatment, to see which approach best reduces spleen size and controls symptoms while being safe for patients with low platelet counts. You may be eligible if: - You are 18 years of age or older - You have been diagnosed with intermediate or high-risk myelofibrosis - You have thrombocytopenia (low platelet count) - You have a noticeably enlarged spleen (more than 5 cm on physical exam) - Your organ function is adequate for treatment You may NOT be eligible if: - You have previously been treated with more than 2 JAK2 inhibitor drugs - You have had more than 6 months of total JAK2 inhibitor treatment - You have had your spleen removed or are planning to - You have active gastrointestinal conditions like Crohn's disease or chronic diarrhea - You have significant, uncontrolled heart disease - You have another active cancer (other than certain limited skin, cervical, prostate, breast, or bladder cancers) - Your life expectancy is less than 6 months Talk to your doctor about whether this trial might be right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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