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Primary MyelofibrosisPhase 3PAC326NCT02055781

Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia

A Phase 3 study testing Pacritinib and Best Available Therapy in people with Primary Myelofibrosis and Post-polycythemia Vera Myelofibrosis, run by CTI BioPharma at 122 sites in 12 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Pacritinib and Best Available Therapy
Could I receive a placebo?
No, everyone receives an active treatment
How many people
311 participants
Where
122 sitesin 12 countries
Study dates
Feb 2014to Apr 2016
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 10Show fewer
  • 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

Who cannot join

  • 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
Show all 9Show fewer
  • 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

Trial locations and contacts

122 sites in 12 countries.

122 sites in 12 countries

United States50 sites

  • New Mexico Cancer Care Alliance

    Albuquerque, New Mexico, 87106

  • St Joseph Mercy Hospital

    Ann Arbor, Michigan, 48106

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Rocky Mountain Cancer Center

    Boulder, Colorado, 80303

  • Northwestern University

    Chicago, Illinois, 60611

  • SCRI-Oncology Hematology Care

    Cincinnati, Ohio, 45242

  • Cleveland Clinic-Taussig Cancer Center

    Cleveland, Ohio, 44195

  • Texas Onocolgy-Baylor Sammons Cancer Center

    Dallas, Texas, 75246

  • UT Southwestern Medical Center

    Dallas, Texas, 75390

  • Henry Ford Health System

    Detroit, Michigan, 48202

  • City of Hope

    Duarte, California, 91010

  • Providence Regional Cancer Partnership

    Everett, Washington, 98201

  • SCRI- Florida Cancer Specialists South Region

    Fort Myers, Florida, 33916

  • UTMB Galveston

    Galveston, Texas, 77555

  • Indiana University Goshen Cancer Centre

    Goshen, Indiana, 46526

  • Green Bay Oncology

    Green Bay, Wisconsin, 54301

  • Upstate Oncology Associates

    Greenville, South Carolina, 29601

  • Hackensack University

    Hackensack, New Jersey, 07601

  • Houston Methodist

    Houston, Texas, 77030

  • University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

  • Investigative Clinical Research of Indiana

    Indianapolis, Indiana, 46260

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • Moores Cancer Centre

    La Jolla, California, 92093

  • Virginia Cancer Specialists

    Leesburg, Virginia, 20176

  • Nebraska Hematology-Oncology, P.C.

    Lincoln, Nebraska, 68506

  • USC Norris Comprehensive Cancer Center

    Los Angeles, California, 90033

  • Norton Cancer Institute, Suburban

    Louisville, Kentucky, 40207

  • Froedtert Hospital and the Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Hematology-Oncology Associates of Northern Jersey

    Morristown, New Jersey, 07962

  • Sarah Cannon Research Institute (SCRI)

    Nashville, Tennessee, 37203

  • Mount Sinai Medical Center

    New York, New York, 10029

  • Columbia University Medical Center

    New York, New York, 10032

  • University of Oklahoma

    Oklahoma City, Oklahoma, 73104

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

  • UPMC Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232

  • Huntsman Cancer Hospital

    Salt Lake City, Utah, 84112

  • Cancer Care Centers of South Texas

    San Antonio, Texas, 78229

  • Mayo Clinic Arizona

    Scottsdale, Arizona, 85259

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98109

  • Siouxland Hematology-Oncology Associates, L.L.P (SHOA)

    Sioux City, Iowa, 51101

  • Providence Cancer Institute

    Southfield, Michigan, 48075

  • Washington University School of Medicine Division of Oncology

    St Louis, Missouri, 63110

  • SCRI - Florida Cancer Specialists North Region

    St. Petersburg, Florida, 33705

  • Stanford Cancer Center

    Stanford, California, 94305

  • Stony Brook University Medical Center

    Stony Brook, New York, 11794

  • H. Lee Moffitt Cancer Center & Research Institute

    Tampa, Florida, 33612

  • Arizona Clinical Research Center

    Tucson, Arizona, 85715

  • Carle Cancer Center

    Urbana, Illinois, 61801

  • George Washington University- Medical Faculty Associates

    Washington D.C., District of Columbia, 20037

Other trials in United States

France12 sites

  • Chu d'Amiens Hopital Sud

    Amiens, Cedex 1, 80054

  • CHU de CAEN

    Caen, 14000

  • Centre Hospitalier de Lens

    Lens, 62300

  • CH de Mulhouse

    Mulhouse, Cedex, 68070

  • Hopital l'Archet, CHU de Nice

    Nice, BP 30 79 06202

  • Hôpital Caremeau

    Nîmes, Cedex 9, 30029

  • Saint Antoine Hospital

    Paris, 75012

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite, 69495

  • CHU Rennes

    Rennes, Cedex 9, 35033

  • CHU de Strasbourg

    Strasbourg, 67091

  • CHU Purpan

    Toulouse, Cedex 9, 31059

  • Institut Gustave Roussy

    Villejuif, 94805

Other trials in France

Germany10 sites

  • Charite-Medical University

    Berlin, 12203

  • Klinik I fur Innere Medizin, Universitat Koln

    Cologne, 50924

  • Gemeinschaftspraxis Hämatologie/Onkologie

    Dresden, 01307

  • University Hospital Essen

    Essen, D-45122

  • Uniklinik Freiburg

    Freiburg im Breisgau, 79106

  • Universitatsklinikum Halle (Saale)

    Halle, 06120

  • University Hospital Leipzig

    Leipzig, 04103

  • Städtisches Klinikum München GmbH

    München, 81737

  • University of Munster

    Münster, 48149

  • University Hospital Ulm

    Ulm, 89081

Other trials in Germany

United Kingdom10 sites

  • Belfast Health and Social Care Trust

    Belfast, Northern Ireland, BT9 7AB

  • Birmingham Heartlands Hospital

    Birmingham, B9 5SS

  • Beatson West of Scotland Cancer Centre

    Glasgow, G12 OYN

  • Leicester Royal Infirmary

    Leicester, LE1 5WW

  • Royal Liverpool University Hospital

    Liverpool, Merseyside, L7 8XP

  • Guy's Hospital

    London, SE1 9RT

  • Hammersmith Hosp - ICH NHS Trust

    London, W12 OHS

  • The Christie NHS Foundation Trust

    Manchester, M20 4BX

  • Oxford University Hospitals NHS Trust

    Oxford, OX3 7LE

  • Royal Hallamshire Hospital

    Sheffield, S10 2JF

Other trials in United Kingdom

Australia7 sites

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000

  • Box Hill Hospital

    Box Hill, Victoria, 3128

  • Haematology and Oncology Clinics of Australia

    Chermside, 4032

  • Monash Health - Monash Medical Centre

    Clayton, Victoria, 3168

  • St George Hospital

    Kogarah, New South Wales, 2217

  • Perth Blood Institute

    Nedlands, Western Australia, 6009

  • Prince of Wales Hospital

    Randwick, 2031

Other trials in Australia

Belgium7 sites

  • ZNA - Stuivenberg

    Antwerp, 2060

  • AZ Sint Jan Brugge-Oostende AV

    Bruges, 8000

  • Hopital Brugmann

    Brussels, 1020

  • Cliniques Universitaires St-Luc

    Brussels, 1200

  • Centre Hospitalier de Jolimont-Lobbes

    Haine-Saint-Paul, Hainaut

  • St Augustinus

    Wilrijk, 2610

  • UC Louvain

    Yvoir, 5530

Other trials in Belgium

Russia7 sites

  • National Haematology Research Center

    Moscow, 125167

  • Republican Hopsital n.a. V.A. Baranov

    Petrozavodsk, 185019

  • Ryazan Regional Clinical Hospital

    Ryazan, 390039

  • Russian Research Institute of Hematology and Transfusiology

    Saint Petersburg, 191024

  • Military Medical Academy n.a. S.M. Kirov

    Saint Petersburg, 194044

  • Saratov State Medical University

    Saratov, Saratov Oblast, 410028

  • Bashkir State Medical University

    Ufa, Bashkortostan Republic, 450083

Other trials in Russia

Hungary6 sites

  • Semmelweis Egyetem AOK

    Budapest, 1083

  • University of Debrecen, Belgyogyaszati Intezet

    Debrecen, 4032

  • Bekes Megyei Pandy Kalman Korhaz

    Gyula, 5700

  • Kaposi Mór Oktató Kórház

    Kaposvár, 7400

  • SZTE II. sz Belgyogyoszati Klinika es Kardiologiai Kozpont

    Szeged, 6720

  • Jász-Nagykun-Szolnok Megyei Hetényi Géza Kórház-Rendelőint

    Szolnok, 5004

Other trials in Hungary

New Zealand5 sites

  • Auckland District Health Board, Auckland City Hospital

    Auckland, 1023

  • Middlemore Hospital

    Auckland, 1640

  • Canterbury District Health Board

    Christchurch, 8001

  • North Shore Hospital

    Takapuna, 0740

  • CCDHB - Wellington Hospital

    Wellington, 6021

Other trials in New Zealand

Czechia4 sites

  • Fakultní nemocnice Brno

    Brno, NAP, 62500

  • University Hospital Hradec Kralove

    Králová, 500 05

  • Faculty Hospital Olomouc

    Olomouc, NAP, 775 20

  • Fakultní nemocnice Plzeň

    Pilsen, NAP, 30460

Other trials in Czechia

Canada2 sites

  • Saint John Regional Hospital

    Saint John, New Brunswick, E2L 4L2

  • Princess Margaret Cancer Center

    Toronto, Ontario, M5G2M9

Other trials in Canada

Netherlands2 sites

  • University Hospital Maastricht

    Maastricht, 6229 HX

  • Erasmus MC

    Rotterdam, 3015 CE

Other trials in Netherlands

About this study

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

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.

Read the detailed description

Multicenter, randomized, controlled, phase 3 trial comparing the safety and efficacy of pacritinib with that of BAT in patients with thrombocytopenia and primary or secondary myelofibrosis. Approximately 300 eligible patients will be randomized in a 1:1:1 allocation to pacritinib 400 mg dosed QD, pacritinib 200 mg dosed BID, or 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). Spleen volume will be measured by MRI or CT at baseline and every 12 weeks thereafter. An independent radiology facility (IRF), blind to treatment assignments, will measure spleen volumes. Patients will also be followed for safety, Leukemia Free Survival (LFS), Overall Survival (OS), frequency of red blood cell (RBC) and platelet transfusions, and other exploratory endpoints. An Independent Data Monitoring Committee (IDMC) will evaluate the safety of pacritinib.

Being tested
Pacritinib, Best Available Therapy
Keywords
Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis, Primary Myelofibrosis, Polycythemia Vera, Essential Thrombocythemia, Bone Marrow Disease, Hematologic Diseases, Hemorrhagic Disorders, Splenomegaly, Pacritinib, MPN-SAF, MPN-SAF TSS, Anemia, Myeloproliferative Neoplasm, Spleen volume, Thrombocytopenia, SB1518
Medical subject headings
Primary Myelofibrosis, Polycythemia Vera, Thrombocythemia, Essential, Bone Marrow Diseases, Hematologic Diseases, Hemorrhagic Disorders, Splenomegaly, Anemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PacritinibDrug

  • Best Available TherapyDrug

Study groups in NCT02055781
Study groupRoleWhat they receive
Pacritinib, Once DailyExperimentalPacritinib 400 mg QD
Receives: Drug: Pacritinib
Pacritinib, Twice DailyExperimentalPacritinib 200 mg BID
Receives: Drug: Pacritinib
Best Available TherapyActive 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.
Receives: Drug: Best Available Therapy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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).

    Measured: Baseline to Week 24

  2. 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.

    Measured: Baseline to Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Feb 2014 (estimated)
  2. Study startFeb 2014
  3. Primary completionApr 2016
  4. Study completionApr 2016

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
311 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02055781
Sponsor's protocol ID
PERSIST-2 (PAC326)
Acronym
PAC326
Status last verified
Oct 2021
First submitted
3 Feb 2014
Last updated
18 Nov 2021
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

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.

    PubMed 29522138

  • Pemmaraju N, Lucchesi A, Oh ST, Wilson K, Hakimi Z, Buckley SA, Suthar P, Parkitny M, Wojciechowski P, Kolonko K, Mascarenhas JO. Efficacy of pacritinib versus momelotinib in patients with thrombocytopenic myelofibrosis: a matching-adjusted indirect treatment comparison. Expert Rev Hematol. 2026 Sep;19(9):1107-1115. doi: 10.1080/17474086.2026.2686813. Epub 2026 Jun 30.

    PubMed 42316774

  • Ajufo 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.

    PubMed 39400386

  • 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.

    PubMed 34551507

  • 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.

    PubMed 33275766

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Pacritinib and Best Available Therapy in people with Primary Myelofibrosis and Post-polycythemia Vera Myelofibrosis, run by CTI BioPharma at 122 sites in 12 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 18 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 122 sites in United States, France, Germany, United Kingdom, Australia and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Nov 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Spleen Volume Reduction (time frame: Baseline to Week 24). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02055781, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2021.

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PAC326 Clinical Trial NCT02055781 | AzTrials