Project Title

Oral Pacritinib Versus Best Available Therapy to Treat Myelofibrosis With Thrombocytopenia (PAC326).

Official Title

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.

Project Summary

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.

The primary objective is to compare the efficacy of two dose-schedule arms(s) of pacritinib (pooled once-daily [QD] and twice-daily [BID] dosing arms) with that of best available therapy (BAT) in patients with thrombocytopenia and primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF). The efficacy co-endpoints for this analysis are the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline to Week 24, as measured by magnetic resonance imaging (MRI) or computed tomography (CT) scan and the proportion of patients achieving a ≥ 50% reduction in total symptom score (TSS) from baseline to Week 24 as measured by the Myeloproliferative Neoplasm Symptom Assessment Form 2.0 (MPN-SAF TSS 2.0).

Blood Disorder

• Primary Myelofibrosis
• Post-polycythemia Vera Myelofibrosis
• Post-essential Thrombocythemia Myelofibrosis

Patient Recruitment Details

Patient recruitment status: Closed

Number of Patients (globally)

311 participants.

Back to Archived Projects...