Oral Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

This study is currently recruiting participants.
Verified December 2013 by Cell Therapeutics
Sponsor:
Information provided by (Responsible Party):
Cell Therapeutics
ClinicalTrials.gov Identifier:
NCT01773187
First received: January 18, 2013
Last updated: December 5, 2013
Last verified: December 2013
  Purpose

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

  Eligibility

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

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

Contacts
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
Investigators
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:
Myelofibrosis
Post-Polycythemia Vera Myelofibrosis
Post-Essential Thrombocythemia Myelofibrosis
Primary Myelofibrosis
Polycythemia
Polycythemia Vera
Thrombocythemia, Essential
Thrombocythemia
Myeloproliferative Disorders
Bone Marrow Disease
Hematologic Diseases
Blood Platelet Disorders
Hemorrhagic Disorders
Splenomegaly
Pacritinib
MPN-SAF
MPN-SAF TSS
Anemia
Myeloproliferative
Myeloproliferative Neoplasm
Spleen
Spleen volume
Thrombocytopenia
SB1518

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

ClinicalTrials.gov processed this record on April 16, 2014