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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01773187




Registration number
NCT01773187
Ethics application status
Date submitted
18/01/2013
Date registered
23/01/2013
Date last updated
29/09/2020

Titles & IDs
Public title
Pacritinib Versus Best Available Therapy to Treat Myelofibrosis
Scientific 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
Secondary ID [1] 0 0
PERSIST-1 (PAC325)
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Myelofibrosis 0 0
Post-polycythemia Vera Myelofibrosis 0 0
Post-essential Thrombocythemia Myelofibrosis 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Experimental: Pacritinib - Pacritinib 400 mg QD

Active comparator: Best Available Therapy - BAT includes any physician-selected treatment for primary or secondary myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases)
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Spleen Volume Reduction
Timepoint [1] 0 0
Baseline to Week 24
Secondary outcome [1] 0 0
Total Symptom Score (TSS) Reduction
Timepoint [1] 0 0
Baseline to Week 24

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/01/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/04/2016
Sample size
Target
Accrual to date
Final
327
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
CTI Investigational Site 61006 - Box Hill
Recruitment hospital [2] 0 0
CTI Investigational Site 61001 - Coffs Harbour
Recruitment hospital [3] 0 0
CTI Investigational Site 61005 - Geelong
Recruitment hospital [4] 0 0
CTI Investigational Site 61003 - Gosford
Recruitment hospital [5] 0 0
CTI Investigational Site 61004 - Hobart
Recruitment hospital [6] 0 0
CTI Investigational Site 61002 - Milton
Recruitment postcode(s) [1] 0 0
- Box Hill
Recruitment postcode(s) [2] 0 0
- Coffs Harbour
Recruitment postcode(s) [3] 0 0
- Geelong
Recruitment postcode(s) [4] 0 0
- Gosford
Recruitment postcode(s) [5] 0 0
- Hobart
Recruitment postcode(s) [6] 0 0
- Milton
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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United States of America
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Nebraska
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United States of America
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New Jersey
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South Carolina
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Belgium
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Antwerp
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Belgium
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Brugge
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Belgium
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Bruxelles
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Belgium
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La Louviere
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Czechia
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Brno
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Czechia
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Olomouc
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Czechia
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Plzen
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Czechia
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Prague
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France
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Amiens
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France
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Caen
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France
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Grenoble
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France
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Lens
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France
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Lille
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France
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Nimes Cedex
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France
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Paris
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France
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Pessac
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France
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Pierre Benite
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France
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Strasbourg
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France
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Toulouse
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Germany
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Berlin
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Dresden
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Essen
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Freiburg
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Koln
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Mainz
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Munchen
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Munster
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Budapest
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Debrecen
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Gyula
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Kaposvar
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Kecskemet
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Szeged
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Szolnok
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Szombathely
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Bologna
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Firenze
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Milano
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Italy
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Monza
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Italy
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Padova
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Italy
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Reggio Emilia
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Italy
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Rimini
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Netherlands
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Amsterdam
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Maastricht
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Rotterdam
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Utrecht
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New Zealand
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Christchurch
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New Zealand
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Dunedin
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New Zealand
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Hamilton
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New Zealand
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Takapuna
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Russian Federation
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Izhevsk
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Moscow
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Petrozavodsk
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Saint Petersburg
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Samara
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Sochi
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St. Petersburg
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Volgograd
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United Kingdom
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Birmingham
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Bournemouth
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Cambridge
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Cardiff
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London
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Manchester
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Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
CTI BioPharma
Address
Country

Ethics approval
Ethics application status

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 primary or secondary myelofibrosis.
Trial website
https://clinicaltrials.gov/study/NCT01773187
Trial related presentations / publications
Mesa RA, Vannucchi AM, Mead A, Egyed M, Szoke A, Suvorov A, Jakucs J, Perkins A, Prasad R, Mayer J, Demeter J, Ganly P, Singer JW, Zhou H, Dean JP, Te Boekhorst PA, Nangalia J, Kiladjian JJ, Harrison CN. Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial. Lancet Haematol. 2017 May;4(5):e225-e236. doi: 10.1016/S2352-3026(17)30027-3. Epub 2017 Mar 20.
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.
Public notes

Contacts
Principal investigator
Name 0 0
Beth Ziemba
Address 0 0
VP, Pharmacovigilance, Clinical Operations, QA
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01773187