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

Arm | Intervention/treatment |
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Experimental: Pacritinib, Once Daily
Pacritinib 400 mg taken orally, once daily
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Drug: Pacritinib |
Experimental: Pacritinib, Twice Daily
Pacritinib 200 mg taken orally, twice daily
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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.
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Drug: Best Available Therapy |
- 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.
- 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.
- 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.

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

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

Study Director: | Mary Campbell, MD | CTI BioPharma |
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 |
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 |
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 |