Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia (PAC326)

October 21, 2021 updated by: CTI BioPharma

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

311

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Chermside, Australia, 4032
        • Haematology and Oncology Clinics of Australia
      • Randwick, Australia, 2031
        • Prince of Wales Hospital
    • New South Wales
      • Kogarah, New South Wales, Australia, 2217
        • St George Hospital
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • Royal Adelaide Hospital
    • Victoria
      • Box Hill, Victoria, Australia, 3128
        • Box Hill Hospital
      • Clayton, Victoria, Australia, 3168
        • Monash Health - Monash Medical Centre
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Perth Blood Institute
  • Belgium
      • Antwerpen, Belgium, 2060
        • ZNA - Stuivenberg
      • Brugge, Belgium, 8000
        • AZ Sint Jan Brugge-Oostende av
      • Brussels, Belgium, 1020
        • Hôpital Brugmann
      • Bruxelles, Belgium, 1200
        • Cliniques Universitaires St-Luc
      • Wilrijk, Belgium, 2610
        • St Augustinus
      • Yvoir, Belgium, 5530
        • UC Louvain
    • Hainaut
      • Haine-Saint-Paul, Hainaut, Belgium
        • Centre Hospitalier de Jolimont-Lobbes
  • Canada
    • New Brunswick
      • Saint John, New Brunswick, Canada, E2L 4L2
        • Saint John Regional Hospital
    • Ontario
      • Toronto, Ontario, Canada, M5G2M9
        • Princess Margaret Cancer Center
  • Czechia
      • Králová, Czechia, 500 05
        • University Hospital Hradec Kralove
    • NAP
      • Brno, NAP, Czechia, 62500
        • Fakultni Nemocnice Brno
      • Olomouc, NAP, Czechia, 775 20
        • Faculty Hospital Olomouc
      • Plzeň, NAP, Czechia, 30460
        • Fakultni nemocnice Plzen
  • France
      • Caen, France, 14000
        • CHU de Caen
      • Lens, France, 62300
        • Centre Hospitalier de LENS
      • Nice, France, BP 30 79 06202
        • Hopital l'Archet, CHU de Nice
      • Paris, France, 75012
        • Saint Antoine Hospital
      • Pierre Benite, France, 69495
        • Centre Hospitalier Lyon Sud
      • Strasbourg, France, 67091
        • CHU de Strasbourg
      • Villejuif Cedex, France, 94805
        • Institut Gustave Roussy
    • Cedex
      • Mulhouse, Cedex, France, 68070
        • CH de Mulhouse
    • Cedex 1
      • Amiens, Cedex 1, France, 80054
        • CHU D'amiens Hôpital Sud
    • Cedex 9
      • Nimes, Cedex 9, France, 30029
        • Hopital Caremeau
      • Rennes, Cedex 9, France, 35033
        • Chu Rennes
      • Toulouse, Cedex 9, France, 31059
        • CHU Purpan
  • Germany
      • Berlin, Germany, 12203
        • Charite-Medical University
      • Dresden, Germany, 01307
        • Gemeinschaftspraxis Hämatologie/Onkologie
      • Essen, Germany, D-45122
        • University Hospital Essen
      • Freiburg, Germany, 79106
        • Uniklinik Freiburg
      • Halle (Saale), Germany, 06120
        • Universitatsklinikum Halle (Saale)
      • Koln, Germany, 50924
        • Klinik I fur Innere Medizin, Universitat Koln
      • Leipzig, Germany, 04103
        • University Hospital Leipzig
      • Munchen, Germany, 81737
        • Städtisches Klinikum München GmbH
      • Munster, Germany, 48149
        • University of Münster
      • Ulm, Germany, 89081
        • University Hospital Ulm
  • Hungary
      • Budapest, Hungary, 1083
        • Semmelweis Egyetem AOK
      • Debrecen, Hungary, 4032
        • University of Debrecen, Belgyogyaszati Intezet
      • Gyula, Hungary, 5700
        • Bekes Megyei Pandy Kalman Korhaz
      • Kaposvár, Hungary, 7400
        • Kaposi Mór Oktató Kórház
      • Szeged, Hungary, 6720
        • SZTE II. sz Belgyogyoszati Klinika es Kardiologiai Kozpont
      • Szolnok, Hungary, 5004
        • Jász-Nagykun-Szolnok Megyei Hetényi Géza Kórház-Rendelőint
  • Netherlands
      • Maastricht, Netherlands, 6229 HX
        • University Hospital Maastricht
      • Rotterdam, Netherlands, 3015 CE
        • Erasmus MC
  • New Zealand
      • Auckland, New Zealand, 1640
        • Middlemore Hospital
      • Auckland, New Zealand, 1023
        • Auckland District Health Board, Auckland City Hospital
      • Christchurch, New Zealand, 8001
        • Canterbury District Health Board
      • Takapuna, New Zealand, 0740
        • North Shore Hospital
      • Wellington, New Zealand, 6021
        • CCDHB - Wellington Hospital
  • Russian Federation
      • Moscow, Russian Federation, 125167
        • National Haematology Research Center
      • Petrozavodsk, Russian Federation, 185019
        • Republican Hopsital n.a. V.A. Baranov
      • Ryazan, Russian Federation, 390039
        • Ryazan Regional Clinical Hospital
      • St. Petersburg, Russian Federation, 191024
        • Russian Research Institute of Hematology and Transfusiology
      • St. Petersburg, Russian Federation, 194044
        • Military Medical Academy n.a. S.M. Kirov
    • Republic Of Bashkortostan
      • Ufa, Republic Of Bashkortostan, Russian Federation, 450083
        • Bashkir State Medical University
    • Saratov Region
      • Saratov, Saratov Region, Russian Federation, 410028
        • Saratov State Medical University
  • United Kingdom
      • Birmingham, United Kingdom, B9 5SS
        • Birmingham Heartlands Hospital
      • Glasgow, United Kingdom, G12 OYN
        • Beatson West of Scotland Cancer Centre
      • Leicester, United Kingdom, LE1 5WW
        • Leicester Royal Infirmary
      • London, United Kingdom, SE1 9RT
        • Guy's Hospital
      • London, United Kingdom, W12 OHS
        • Hammersmith Hosp - ICH NHS Trust
      • Manchester, United Kingdom, M20 4BX
        • The Christie NHS Foundation Trust
      • Oxford, United Kingdom, OX3 7LE
        • Oxford University Hospitals NHS Trust
      • Sheffield, United Kingdom, S10 2JF
        • Royal Hallamshire Hospital
    • Merseyside
      • Liverpool, Merseyside, United Kingdom, L7 8XP
        • Royal Liverpool University Hospital
    • N. Ireland
      • Belfast, N. Ireland, United Kingdom, BT9 7AB
        • Belfast Health and Social Care Trust
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85259
        • Mayo Clinic Arizona
      • Tucson, Arizona, United States, 85715
        • Arizona Clinical Research Center
    • California
      • Duarte, California, United States, 91010
        • City of Hope
      • La Jolla, California, United States, 92093
        • Moores Cancer Centre
      • Los Angeles, California, United States, 90033
        • Usc Norris Comprehensive Cancer Center
      • Stanford, California, United States, 94305
        • Stanford Cancer Center
    • Colorado
      • Boulder, Colorado, United States, 80303
        • Rocky Mountain Cancer Center
    • District of Columbia
      • Washington, District of Columbia, United States, 20037
        • George Washington University- Medical Faculty Associates
    • Florida
      • Fort Myers, Florida, United States, 33916
        • SCRI- Florida Cancer Specialists South Region
      • Saint Petersburg, Florida, United States, 33705
        • SCRI - Florida Cancer Specialists North Region
      • Tampa, Florida, United States, 33612
        • H. Lee Moffitt Cancer Center & Research Institute
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
      • Urbana, Illinois, United States, 61801
        • Carle Cancer Center
    • Indiana
      • Goshen, Indiana, United States, 46526
        • Indiana University Goshen Cancer Centre
      • Indianapolis, Indiana, United States, 46260
        • Investigative Clinical Research of Indiana
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
      • Sioux City, Iowa, United States, 51101
        • Siouxland Hematology-Oncology Associates, L.L.P (SHOA)
    • Kentucky
      • Louisville, Kentucky, United States, 40207
        • Norton Cancer Institute, Suburban
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
      • Ann Arbor, Michigan, United States, 48106
        • St Joseph Mercy Hospital
      • Detroit, Michigan, United States, 48202
        • Henry Ford Health System
      • Southfield, Michigan, United States, 48075
        • Providence Cancer Institute
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine Division of Oncology
    • Nebraska
      • Lincoln, Nebraska, United States, 68506
        • Nebraska Hematology-Oncology, P.C.
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack University
      • Morristown, New Jersey, United States, 07962
        • Hematology-Oncology Associates of Northern Jersey
    • New Mexico
      • Albuquerque, New Mexico, United States, 87106
        • New Mexico Cancer Care Alliance
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center
      • New York, New York, United States, 10029
        • Mount Sinai Medical Center
      • Stony Brook, New York, United States, 11794
        • Stony Brook University Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45242
        • SCRI-Oncology Hematology Care
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic-Taussig Cancer Center
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • University of Oklahoma
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Thomas Jefferson University
      • Pittsburgh, Pennsylvania, United States, 15232
        • UPMC Hillman Cancer Center
    • South Carolina
      • Greenville, South Carolina, United States, 29601
        • Upstate Oncology Associates
    • Tennessee
      • Nashville, Tennessee, United States, 37203
        • Sarah Cannon Research Institute (SCRI)
    • Texas
      • Dallas, Texas, United States, 75390
        • UT Southwestern Medical Center
      • Dallas, Texas, United States, 75246
        • Texas Onocolgy-Baylor Sammons Cancer Center
      • Galveston, Texas, United States, 77555
        • UTMB Galveston
      • Houston, Texas, United States, 77030
        • University of Texas MD Anderson Cancer Center
      • Houston, Texas, United States, 77030
        • Houston Methodist
      • San Antonio, Texas, United States, 78229
        • Cancer Care Centers of South Texas
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Huntsman Cancer Hospital
    • Virginia
      • Leesburg, Virginia, United States, 20176
        • Virginia Cancer Specialists
    • Washington
      • Everett, Washington, United States, 98201
        • Providence Regional Cancer Partnership
      • Seattle, Washington, United States, 98109
        • Fred Hutchinson Cancer Research Center
    • Wisconsin
      • Green Bay, Wisconsin, United States, 54301
        • Green Bay Oncology
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Froedtert Hospital and the Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Pacritinib, Once Daily
Pacritinib 400 mg QD
Drug: Pacritinib
Experimental: Pacritinib, Twice Daily
Pacritinib 200 mg BID
Drug: Pacritinib
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.
Drug: Best Available Therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Spleen Volume Reduction
Time Frame: Baseline to Week 24
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
Time Frame: Baseline to Week 24
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CTI BioPharma

Investigators

  • Study Director: Simran Singh, Sr. Director, Head of Clinical Operations

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 1, 2014

Primary Completion (Actual)

April 1, 2016

Study Completion (Actual)

April 1, 2016

Study Registration Dates

First Submitted

February 3, 2014

First Submitted That Met QC Criteria

February 4, 2014

First Posted (Estimate)

February 5, 2014

Study Record Updates

Last Update Posted (Actual)

November 18, 2021

Last Update Submitted That Met QC Criteria

October 21, 2021

Last Verified

October 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PERSIST-2 (PAC326)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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