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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02055781
Registration number
NCT02055781
Ethics application status
Date submitted
3/02/2014
Date registered
5/02/2014
Date last updated
18/11/2021
Titles & IDs
Public title
Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia
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Scientific 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
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Secondary ID [1]
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PERSIST-2 (PAC326)
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Universal Trial Number (UTN)
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Trial acronym
PAC326
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Primary Myelofibrosis
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Post-polycythemia Vera Myelofibrosis
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Post-essential Thrombocythemia Myelofibrosis
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Condition category
Condition code
Blood
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Haematological diseases
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Blood
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Other blood disorders
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Musculoskeletal
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Other muscular and skeletal disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Cancer
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Leukaemia - Chronic leukaemia
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Experimental: Pacritinib, Once Daily - Pacritinib 400 mg QD
Experimental: Pacritinib, Twice Daily - Pacritinib 200 mg BID
Active comparator: Best Available Therapy - BAT includes any physician-selected treatment for myelofibrosis, such as approved JAK2 inhibitors administered according to package insert for patients with thrombocytopenia, and may include any treatment received before study entry.
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Spleen Volume Reduction
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Assessment method [1]
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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).
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Timepoint [1]
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Baseline to Week 24
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Primary outcome [2]
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Total Symptom Score (TSS) Reduction
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Assessment method [2]
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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 (MPN-SAF TSS 2.0). Responses (on a scale from 0 \[absent\] to 10 \[worst imaginable\]) to questions about symptoms (tiredness, early satiety, abdominal discomfort, night sweats, pruritus, bone pain, and pain under the ribs on the left side) were used to calculate the TSS.
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Timepoint [2]
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Baseline to Week 24
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Eligibility
Key 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
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Minimum age
18
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/02/2014
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Date of last participant enrolment
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Actual
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Date of last data collection
Anticipated
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Actual
1/04/2016
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Sample size
Target
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Accrual to date
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Final
311
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Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC,WA
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Recruitment hospital [1]
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St George Hospital - Kogarah
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Royal Adelaide Hospital - Adelaide
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Box Hill Hospital - Box Hill
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Monash Health - Monash Medical Centre - Clayton
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Perth Blood Institute - Nedlands
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Haematology and Oncology Clinics of Australia - Chermside
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Prince of Wales Hospital - Randwick
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2217 - Kogarah
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5000 - Adelaide
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3128 - Box Hill
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3168 - Clayton
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6009 - Nedlands
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4032 - Chermside
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2031 - Randwick
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Funding & Sponsors
Primary sponsor type
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Name
CTI BioPharma
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Ethics approval
Ethics application status
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Summary
Brief summary
Phase 3, randomized, controlled study to evaluate the safety and efficacy of oral pacritinib compared to Best Available Therapy (BAT) in patients with thrombocytopenia and primary or secondary myelofibrosis.
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Trial website
https://clinicaltrials.gov/study/NCT02055781
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Trial related presentations / publications
Mascarenhas J, Hoffman R, Talpaz M, Gerds AT, Stein B, Gupta V, Szoke A, Drummond M, Pristupa A, Granston T, Daly R, Al-Fayoumi S, Callahan JA, Singer JW, Gotlib J, Jamieson C, Harrison C, Mesa R, Verstovsek S. Pacritinib vs Best Available Therapy, Including Ruxolitinib, in Patients With Myelofibrosis: A Randomized Clinical Trial. JAMA Oncol. 2018 May 1;4(5):652-659. doi: 10.1001/jamaoncol.2017.5818. Verstovsek S, Mesa R, Talpaz M, Kiladjian JJ, Harrison CN, Oh ST, Vannucchi AM, Rampal R, Scott BL, Buckley SA, Craig AR, Roman-Torres K, Mascarenhas JO. Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia. Haematologica. 2022 Jul 1;107(7):1599-1607. doi: 10.3324/haematol.2021.279415. Tremblay D, Mesa R, Scott B, Buckley S, Roman-Torres K, Verstovsek S, Mascarenhas J. Pacritinib demonstrates spleen volume reduction in patients with myelofibrosis independent of JAK2V617F allele burden. Blood Adv. 2020 Dec 8;4(23):5929-5935. doi: 10.1182/bloodadvances.2020002970.
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Public notes
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Contacts
Principal investigator
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Simran Singh
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Sr. Director, Head of Clinical Operations
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02055781
Download to PDF