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

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ClinicalTrials.gov Identifier: NCT02055781
Recruitment Status : Terminated
First Posted : February 5, 2014
Last Update Posted : October 22, 2018
Sponsor:
Information provided by (Responsible Party):
CTI BioPharma

Study Description
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Brief 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.

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

Detailed Description:
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).
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 311 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : December 2013
Actual Primary Completion Date : August 2016
Actual Study Completion Date : November 2016

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Pacritinib, Once Daily
Pacritinib 400 mg taken orally, once daily
 Drug: Pacritinib
Experimental: Pacritinib, Twice Daily
Pacritinib 200 mg taken orally, twice daily
 Drug: Pacritinib
Active Comparator: Best Available Therapy
Best Available Therapy includes any physician-selected treatment for primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis, such as approved JAK2 inhibitors, and may include any treatment received before study entry. Best Available Therapy may include ruxolitinib, other approved JAK2 inhibitors, hydroxyurea, glucocorticoids, erythropoietic agents, immunomodulatory agents, mercaptopurine, danazol, interferons, cytarabine, melphalan, or other agents and may also include no treatment and symptom-directed treatment without myelofibrosis-specific treatment.
 Drug: Best Available Therapy


Outcome Measures
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Primary Outcome Measures :
  1. To compare the efficacy of two dose-schedule arms of pacritinib (pooled once-daily and twice-daily dosing arms) with that of Best Available Therapy [ Time Frame: Baseline to Week 24 ]
    To compare the efficacy of two dose-schedule arms of pacritinib (pooled once-daily and twice-daily dosing arms) with that of Best Available Therapy in patients with thrombocytopenia and primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis; 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) and the 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.


Secondary Outcome Measures :
  1. To compare the efficacy of once-daily pacritinib with that of Best Available Therapy [ Time Frame: Baseline to Week 24 ]
    To compare the efficacy of once-daily pacritinib with that of Best Available Therapy, as assessed by 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) and the proportion of patients achieving a ≥ 50% reduction in the total symptom score from baseline to Week 24 on the Myeloproliferative Neoplasm Symptom Assessment Form 2.0.

  2. To compare the efficacy of twice-daily pacritinib with that of Best Available Therapy [ Time Frame: Baseline to Week 24 ]
    To compare the efficacy of twice-daily pacritinib with that of Best Available Therapy, as assessed by 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) and the proportion of patients achieving a ≥ 50% reduction in the total symptom score from baseline to Week 24 on the myeloproliferate Neoplasm Symptom Assessment Form 2.0.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Intermediate -1 or -2 or high-risk Myelofibrosis (per Passamonti et al 2010)
  • Thrombocytopenia (platelet count ≤ 100,000/µL) at any time after signing informed consent
  • 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
  • At least 6 months from prior splenic irradiation
  • At least 1-4 weeks since prior myelofibrosis therapy, including any erythropoietic or thrombopoietic agent
  • Not pregnant, not lactating, and agree to use effective birth control
  • Able and willing to undergo frequent MRI or CT assessments and complete symptom assessments using a patient-reported outcome instrument

Exclusion Criteria:

  • Prior treatment with more than 2 JAK2 inhibitors or with pacritinib
  • There is no maximum cumulative prior JAK2 inhibitor treatment
  • 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
  • Active bleeding that requires hospitalization during the screening period
  • 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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02055781


Locations
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Sponsors and Collaborators
CTI BioPharma
Investigators
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Study Director: Mary Campbell, MD CTI BioPharma
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: CTI BioPharma
ClinicalTrials.gov Identifier: NCT02055781    
Other Study ID Numbers: PERSIST-2 (PAC326)
First Posted: February 5, 2014    Key Record Dates
Last Update Posted: October 22, 2018
Last Verified: October 2018
Keywords provided by CTI BioPharma:
Pacritinib
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
MPN-SAF
MPN-SAF TSS
Anemia
Myeloproliferative
Myeloproliferative Neoplasm
Spleen
Spleen volume
Thrombocytopenia
SB1518
Additional relevant MeSH terms:
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Polycythemia Vera
Primary Myelofibrosis
Thrombocytopenia
Polycythemia
Thrombocytosis
Thrombocythemia, Essential
Myeloproliferative Disorders
Bone Marrow Diseases
 Hematologic Diseases
Blood Platelet Disorders
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Neoplasms
Blood Coagulation Disorders
Hemorrhagic Disorders