Oral Pacritinib Versus Best Available Therapy to Treat Myelofibrosis - Full Text View

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 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:

Genetics Home Reference related topics: essential thrombocythemia polycythemia vera primary myelofibrosis

Genetic and Rare Diseases Information Center resources: Essential Thrombocythemia Myelofibrosis Chronic Myeloproliferative Disorders Splenomegaly Polycythemia Vera

U.S. FDA Resources

Further study details as provided by CTI BioPharma:

Primary Outcome Measures:

Efficacy [ Time Frame: Baseline to Week 24 ]
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 ]
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)


Enrollment:	327
Study Start Date:	December 2012
Study Completion Date:	June 2016
Primary Completion Date:	January 2015 (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 (Adult, Senior)
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)
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01773187

Show 81 Study Locations

Sponsors and Collaborators

CTI BioPharma

Investigators


Study Director:	James Dean, MD, PhD	CTI BioPharma

More Information


Responsible Party:	CTI BioPharma
ClinicalTrials.gov Identifier:	NCT01773187 History of Changes
Other Study ID Numbers:	PERSIST-1 (PAC325)
Study First Received:	January 18, 2013
Last Updated:	December 15, 2016

Keywords provided by CTI BioPharma:


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 Thrombocytosis Thrombocythemia, Essential Myeloproliferative Disorders	Bone Marrow Diseases Hematologic Diseases Blood Platelet Disorders Blood Coagulation Disorders Hemorrhagic Disorders

ClinicalTrials.gov processed this record on July 17, 2017