NCT02055781

Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia

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

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 311 (actual)
• Sponsor: CTI BioPharma · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Phase 3, randomized, controlled study to evaluate the safety and efficacy of oral pacritinib compared to Best Available Therapy (BAT) in patients with thrombocytopenia and primary or secondary myelofibrosis.

Detailed description

Multicenter, randomized, controlled, phase 3 trial comparing the safety and efficacy of pacritinib with that of BAT in patients with thrombocytopenia and primary or secondary myelofibrosis. Approximately 300 eligible patients will be randomized in a 1:1:1 allocation to pacritinib 400 mg dosed QD, pacritinib 200 mg dosed BID, or BAT (includes any physician-selected treatment for myelofibrosis, such as approved JAK2 inhibitors administered according to package insert for patients with thrombocytopenia, and may include any treatment received before study entry). Spleen volume will be measured by MRI or CT at baseline and every 12 weeks thereafter. An independent radiology facility (IRF), blind to treatment assignments, will measure spleen volumes. Patients will also be followed for safety, Leukemia Free Survival (LFS), Overall Survival (OS), frequency of red blood cell (RBC) and platelet transfusions, and other exploratory endpoints. An Independent Data Monitoring Committee (IDMC) will evaluate the safety of pacritinib.

Conditions

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

Interventions

Type	Name	Description
DRUG	Pacritinib
DRUG	Best Available Therapy

Timeline

• Start date: 2014-02-01
• Primary completion: 2016-04-01
• Completion: 2016-04-01
• First posted: 2014-02-05
• Last updated: 2021-11-18
• Results posted: 2021-11-18

Locations

122 sites across 12 countries: United States, Australia, Belgium, Canada, Czechia, France, Germany, Hungary, Netherlands, New Zealand, Russia, United Kingdom

Regulatory

• FDA-regulated drug study