Oral Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

This study is currently recruiting participants.
Verified December 2013 by Cell Therapeutics
Information provided by (Responsible Party):
Cell Therapeutics
ClinicalTrials.gov Identifier:
First received: January 18, 2013
Last updated: December 5, 2013
Last verified: December 2013

The primary hypothesis of the study is that treatment with pacritinib results in a greater proportion of patients achieving ≥ 35% reduction in spleen volume from baseline to Week 24 than treatment with BAT.

Condition Intervention Phase
Primary Myelofibrosis
Post-polycythemia Vera Myelofibrosis
Post-essential Thrombocythemia Myelofibrosis
Drug: Pacritinib
Drug: Best Available Therapy
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Randomized Controlled Phase 3 Study of Oral Pacritinib Versus Best Available Therapy in Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis

Resource links provided by NLM:

Further study details as provided by Cell Therapeutics:

Primary Outcome Measures:
  • Efficacy [ Time Frame: Baseline to Week 24 ] [ Designated as safety issue: No ]
    To compare the efficacy of pacritinib with that of best available therapy (BAT) in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF); the efficacy measure for this analysis is the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline to week 24 by magnetic resonance imaging (MRI) or computed tomography (CT)

Secondary Outcome Measures:
  • Symptomatic Efficacy [ Time Frame: Baseline to week 24 ] [ Designated as safety issue: No ]
    To compare pacritinib with best available therapy with respect to the proportion of patients with >= 50% reduction in total score from baseline to week 24 on the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF TSS)

Estimated Enrollment: 270
Study Start Date: December 2012
Estimated Study Completion Date: August 2017
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Pacritinib
Pacritinib 400 mg taken orally, once daily
Drug: Pacritinib
Active Comparator: Best Available Therapy
BAT includes any physician-selected treatment for PMF, PPV-MF, or PET-MF with the exclusion of JAK inhibitors (inhibitors of Janus kinases). For example, BAT may include hydroxyurea, glucocorticoids, erythropoietic agents, immunomodulatory agents, mercaptopurine, danazol, interferons, cytarabine, melphalan, or other agents.
Drug: Best Available Therapy


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Intermediate -1 or -2 or high-risk Myelofibrosis (per Passamonti et al 2010)
  • Palpable splenomegaly ≥ 5 cm on physical examination
  • Total Symptom Score >13 on the MPN-SAF TSS 2.0, not including the inactivity question
  • Patients who are platelet or red blood cell transfusion-dependent are eligible
  • Adequate white blood cell counts (with low blast counts), liver function, and renal function
  • No spleen radiation therapy for 6-12 months
  • Last therapy for myelofibrosis was 2-4 weeks ago, including any erythropoietic or thrombopoietic agent
  • Not pregnant, not lactating, and agree to use effective birth control

Exclusion Criteria:

  • Prior treatment with a JAK2 inhibitor
  • History of (or plans to undergo) spleen removal surgery or allogeneic stem cell transplant
  • Ongoing gastrointestinal medical condition such as Crohn's disease, Inflammatory bowel disease, chronic diarrhea, or constipation
  • Cardiovascular disease, including recent history or currently clinically symptomatic and uncontrolled: congestive heart failure, arrhythmia, angina, QTc prolongation or other QTc risk factors, myocardial infarction
  • Other malignancy within last 3 years other than certain limited skin, cervical, prostate, breast, or bladder cancers
  • Other ongoing, uncontrolled illnesses (including HIV infection and active hepatitis A, B, or C), psychiatric disorder, or social situation that would prevent good care on this study
  • Life expectancy < 6 months
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01773187

Contact: Valentina Zhukova-Harrill, MD +44 (0) 131-200-6320
Contact: Gordon Thomson, PM +44 (0) 131-440-6441 gthomson@ockham.com

  Show 73 Study Locations
Sponsors and Collaborators
Cell Therapeutics
Study Director: James Dean, MD, PhD Cell Therapeutics
  More Information

No publications provided

Responsible Party: Cell Therapeutics
ClinicalTrials.gov Identifier: NCT01773187     History of Changes
Other Study ID Numbers: PERSIST-1 (PAC325)
Study First Received: January 18, 2013
Last Updated: December 5, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Cell Therapeutics:
Blood Platelet Disorders
Post-Polycythemia Vera Myelofibrosis
Post-Essential Thrombocythemia Myelofibrosis
Primary Myelofibrosis
Polycythemia Vera
Thrombocythemia, Essential
Myeloproliferative Disorders
Bone Marrow Disease
Hematologic Diseases
Hemorrhagic Disorders
Myeloproliferative Neoplasm
Spleen volume

Additional relevant MeSH terms:
Blood Platelet Disorders
Primary Myelofibrosis
Polycythemia Vera
Thrombocythemia, Essential
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Blood Coagulation Disorders
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on April 17, 2014