Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia
PAC326
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
1 other identifier
interventional
311
12 countries
122
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Feb 2014
122 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2014
CompletedFirst Submitted
Initial submission to the registry
February 3, 2014
CompletedFirst Posted
Study publicly available on registry
February 5, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2016
CompletedResults Posted
Study results publicly available
November 18, 2021
CompletedNovember 18, 2021
October 1, 2021
2.2 years
February 3, 2014
November 23, 2020
October 21, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Spleen Volume Reduction
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).
Baseline to Week 24
Total Symptom Score (TSS) Reduction
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.
Baseline to Week 24
Study Arms (3)
Pacritinib, Once Daily
EXPERIMENTALPacritinib 400 mg QD
Pacritinib, Twice Daily
EXPERIMENTALPacritinib 200 mg BID
Best Available Therapy
ACTIVE COMPARATORBAT 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.
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- CTI BioPharmalead
Study Sites (122)
Mayo Clinic Arizona
Scottsdale, Arizona, 85259, United States
Arizona Clinical Research Center
Tucson, Arizona, 85715, United States
City of Hope
Duarte, California, 91010, United States
Moores Cancer Centre
La Jolla, California, 92093, United States
USC Norris Comprehensive Cancer Center
Los Angeles, California, 90033, United States
Stanford Cancer Center
Stanford, California, 94305, United States
Rocky Mountain Cancer Center
Boulder, Colorado, 80303, United States
George Washington University- Medical Faculty Associates
Washington D.C., District of Columbia, 20037, United States
SCRI- Florida Cancer Specialists South Region
Fort Myers, Florida, 33916, United States
SCRI - Florida Cancer Specialists North Region
St. Petersburg, Florida, 33705, United States
H. Lee Moffitt Cancer Center & Research Institute
Tampa, Florida, 33612, United States
Northwestern University
Chicago, Illinois, 60611, United States
Carle Cancer Center
Urbana, Illinois, 61801, United States
Indiana University Goshen Cancer Centre
Goshen, Indiana, 46526, United States
Investigative Clinical Research of Indiana
Indianapolis, Indiana, 46260, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
Siouxland Hematology-Oncology Associates, L.L.P (SHOA)
Sioux City, Iowa, 51101, United States
Norton Cancer Institute, Suburban
Louisville, Kentucky, 40207, United States
St Joseph Mercy Hospital
Ann Arbor, Michigan, 48106, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Henry Ford Health System
Detroit, Michigan, 48202, United States
Providence Cancer Institute
Southfield, Michigan, 48075, United States
Washington University School of Medicine Division of Oncology
St Louis, Missouri, 63110, United States
Nebraska Hematology-Oncology, P.C.
Lincoln, Nebraska, 68506, United States
Hackensack University
Hackensack, New Jersey, 07601, United States
Hematology-Oncology Associates of Northern Jersey
Morristown, New Jersey, 07962, United States
New Mexico Cancer Care Alliance
Albuquerque, New Mexico, 87106, United States
Mount Sinai Medical Center
New York, New York, 10029, United States
Columbia University Medical Center
New York, New York, 10032, United States
Stony Brook University Medical Center
Stony Brook, New York, 11794, United States
SCRI-Oncology Hematology Care
Cincinnati, Ohio, 45242, United States
Cleveland Clinic-Taussig Cancer Center
Cleveland, Ohio, 44195, United States
University of Oklahoma
Oklahoma City, Oklahoma, 73104, United States
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232, United States
Upstate Oncology Associates
Greenville, South Carolina, 29601, United States
Sarah Cannon Research Institute (SCRI)
Nashville, Tennessee, 37203, United States
Texas Onocolgy-Baylor Sammons Cancer Center
Dallas, Texas, 75246, United States
UT Southwestern Medical Center
Dallas, Texas, 75390, United States
UTMB Galveston
Galveston, Texas, 77555, United States
Houston Methodist
Houston, Texas, 77030, United States
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030, United States
Cancer Care Centers of South Texas
San Antonio, Texas, 78229, United States
Huntsman Cancer Hospital
Salt Lake City, Utah, 84112, United States
Virginia Cancer Specialists
Leesburg, Virginia, 20176, United States
Providence Regional Cancer Partnership
Everett, Washington, 98201, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109, United States
Green Bay Oncology
Green Bay, Wisconsin, 54301, United States
Froedtert Hospital and the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
St George Hospital
Kogarah, New South Wales, 2217, Australia
Royal Adelaide Hospital
Adelaide, South Australia, 5000, Australia
Box Hill Hospital
Box Hill, Victoria, 3128, Australia
Monash Health - Monash Medical Centre
Clayton, Victoria, 3168, Australia
Perth Blood Institute
Nedlands, Western Australia, 6009, Australia
Haematology and Oncology Clinics of Australia
Chermside, 4032, Australia
Prince of Wales Hospital
Randwick, 2031, Australia
Centre Hospitalier de Jolimont-Lobbes
Haine-Saint-Paul, Hainaut, Belgium
ZNA - Stuivenberg
Antwerp, 2060, Belgium
AZ Sint Jan Brugge-Oostende AV
Bruges, 8000, Belgium
Hopital Brugmann
Brussels, 1020, Belgium
Cliniques Universitaires St-Luc
Brussels, 1200, Belgium
St Augustinus
Wilrijk, 2610, Belgium
UC Louvain
Yvoir, 5530, Belgium
Saint John Regional Hospital
Saint John, New Brunswick, E2L 4L2, Canada
Princess Margaret Cancer Center
Toronto, Ontario, M5G2M9, Canada
Fakultní nemocnice Brno
Brno, NAP, 62500, Czechia
Faculty Hospital Olomouc
Olomouc, NAP, 775 20, Czechia
Fakultní nemocnice Plzeň
Pilsen, NAP, 30460, Czechia
University Hospital Hradec Kralove
Králová, 500 05, Czechia
Chu d'Amiens Hopital Sud
Amiens, Cedex 1, 80054, France
Hôpital Caremeau
Nîmes, Cedex 9, 30029, France
CHU Rennes
Rennes, Cedex 9, 35033, France
CHU Purpan
Toulouse, Cedex 9, 31059, France
CH de Mulhouse
Mulhouse, Cedex, 68070, France
CHU de CAEN
Caen, 14000, France
Centre Hospitalier de Lens
Lens, 62300, France
Hopital l'Archet, CHU de Nice
Nice, BP 30 79 06202, France
Saint Antoine Hospital
Paris, 75012, France
Centre Hospitalier Lyon Sud
Pierre-Bénite, 69495, France
CHU de Strasbourg
Strasbourg, 67091, France
Institut Gustave Roussy
Villejuif, 94805, France
Charite-Medical University
Berlin, 12203, Germany
Klinik I fur Innere Medizin, Universitat Koln
Cologne, 50924, Germany
Gemeinschaftspraxis Hämatologie/Onkologie
Dresden, 01307, Germany
University Hospital Essen
Essen, D-45122, Germany
Uniklinik Freiburg
Freiburg im Breisgau, 79106, Germany
Universitatsklinikum Halle (Saale)
Halle, 06120, Germany
University Hospital Leipzig
Leipzig, 04103, Germany
Städtisches Klinikum München GmbH
München, 81737, Germany
University of Munster
Münster, 48149, Germany
University Hospital Ulm
Ulm, 89081, Germany
Semmelweis Egyetem AOK
Budapest, 1083, Hungary
University of Debrecen, Belgyogyaszati Intezet
Debrecen, 4032, Hungary
Bekes Megyei Pandy Kalman Korhaz
Gyula, 5700, Hungary
Kaposi Mór Oktató Kórház
Kaposvár, 7400, Hungary
SZTE II. sz Belgyogyoszati Klinika es Kardiologiai Kozpont
Szeged, 6720, Hungary
Jász-Nagykun-Szolnok Megyei Hetényi Géza Kórház-Rendelőint
Szolnok, 5004, Hungary
University Hospital Maastricht
Maastricht, 6229 HX, Netherlands
Erasmus MC
Rotterdam, 3015 CE, Netherlands
Auckland District Health Board, Auckland City Hospital
Auckland, 1023, New Zealand
Middlemore Hospital
Auckland, 1640, New Zealand
Canterbury District Health Board
Christchurch, 8001, New Zealand
North Shore Hospital
Takapuna, 0740, New Zealand
CCDHB - Wellington Hospital
Wellington, 6021, New Zealand
Bashkir State Medical University
Ufa, Bashkortostan Republic, 450083, Russia
Saratov State Medical University
Saratov, Saratov Oblast, 410028, Russia
National Haematology Research Center
Moscow, 125167, Russia
Republican Hopsital n.a. V.A. Baranov
Petrozavodsk, 185019, Russia
Ryazan Regional Clinical Hospital
Ryazan, 390039, Russia
Russian Research Institute of Hematology and Transfusiology
Saint Petersburg, 191024, Russia
Military Medical Academy n.a. S.M. Kirov
Saint Petersburg, 194044, Russia
Royal Liverpool University Hospital
Liverpool, Merseyside, L7 8XP, United Kingdom
Belfast Health and Social Care Trust
Belfast, Northern Ireland, BT9 7AB, United Kingdom
Birmingham Heartlands Hospital
Birmingham, B9 5SS, United Kingdom
Beatson West of Scotland Cancer Centre
Glasgow, G12 OYN, United Kingdom
Leicester Royal Infirmary
Leicester, LE1 5WW, United Kingdom
Guy's Hospital
London, SE1 9RT, United Kingdom
Hammersmith Hosp - ICH NHS Trust
London, W12 OHS, United Kingdom
The Christie NHS Foundation Trust
Manchester, M20 4BX, United Kingdom
Oxford University Hospitals NHS Trust
Oxford, OX3 7LE, United Kingdom
Royal Hallamshire Hospital
Sheffield, S10 2JF, United Kingdom
Related Publications (4)
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.
PMID: 29522138BACKGROUNDAjufo H, Bewersdorf JP, Harrison C, Palandri F, Mascarenhas J, Palmer J, Gerds A, Kiladjian JJ, Buckley S, Derkach A, Roman-Torres K, Rampal RK. Pacritinib Response Is Associated With Overall Survival in Myelofibrosis: PERSIST-2 Landmark Analysis of Survival. Eur J Haematol. 2025 Feb;114(2):238-247. doi: 10.1111/ejh.14321. Epub 2024 Oct 14.
PMID: 39400386DERIVEDVerstovsek 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.
PMID: 34551507DERIVEDTremblay 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.
PMID: 33275766DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Beth Ziemba
- Organization
- CTI BioPharma Corp.
Study Officials
- STUDY DIRECTOR
Simran Singh
Sr. Director, Head of Clinical Operations
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 3, 2014
First Posted
February 5, 2014
Study Start
February 1, 2014
Primary Completion
April 1, 2016
Study Completion
April 1, 2016
Last Updated
November 18, 2021
Results First Posted
November 18, 2021
Record last verified: 2021-10