A Phase 3 Study of Pacritinib in Patients with Myelofibrosis
Research type
Research Study
Full 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
IRAS ID
150545
Contact name
Adam Mead
Contact email
Sponsor organisation
Cell Therapeutics, Inc.
Eudract number
2013-004000-19
Clinicaltrials.gov Identifier
Research summary
This study is a multicenter, randomized, controlled, phase 3 study. It will compare the efficacy and safety of two dose schedules of pacritinib in pooled and individual group analyses versus Best Available Therapy (BAT) in patients with thrombocytopenia and PMF, PPV-MF, or PET-MF. A total of 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.
Spleen volume will be measured by MRI or CT at baseline and every 12 weeks thereafter. The analysis of the primary outcome of spleen response will be performed when all randomized patients have completed the Week 24 MRI or CT evaluation, met progressive disease criteria, or discontinued study treatment,whichever occurs first.
An independent radiology facility (IRF), blind to treatment assignments, will measure spleen volumes.
Approximately 100 global centers are planned to enroll patients in hospital sites over an estimated period of 11 months and randomise approximately 300 patients.
Patients will be enrolled over a 12 month enrollment period and patients will be assessed weekly for the first month, then monthly to week 24. After week 24, the patients will be assessed every 12 weeks for a further 3 years. Patients will be assessed by measuring spleen volume by Magnetic Resonance Scanning (MRI) or by Computerised Tomography (CT) every 12 weeks.
REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
14/EM/0186
Date of REC Opinion
30 May 2014
REC opinion
Further Information Favourable Opinion