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

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ClinicalTrials.gov Identifier: NCT02055781
Recruitment Status : Terminated
First Posted : February 5, 2014
Last Update Posted : October 22, 2018
Sponsor:
Information provided by (Responsible Party):
CTI BioPharma

Brief Summary:
The primary hypothesis of the study is that treatment with either once-daily or twice-daily pacritinib results in a greater proportion of patients with thrombocytopenia and myelofibrosis achieving ≥ 35% reduction in spleen volume from baseline to Week 24 than treatment with Best Available Therapy, and a greater proportion of patients achieving a ≥ 50% reduction in total symptom score from baseline to Week 24 as measured by the Myeloproliferative Neoplasm Symptom Assessment Form 2.0.

Condition or disease Intervention/treatment Phase
Primary Myelofibrosis Post-polycythemia Vera Myelofibrosis Post-essential Thrombocythemia Myelofibrosis Drug: Pacritinib Drug: Best Available Therapy Phase 3

Detailed Description:
The primary objective is to compare the efficacy of two dose-schedule arms(s) of pacritinib (pooled once-daily [QD] and twice-daily [BID] dosing arms) with that of best available therapy (BAT) in patients with thrombocytopenia and primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF). The efficacy co-endpoints for this analysis are the 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) scan and the proportion of patients achieving a ≥ 50% reduction in total symptom score (TSS) from baseline to Week 24 as measured by the Myeloproliferative Neoplasm Symptom Assessment Form 2.0 (MPN-SAF TSS 2.0).

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 311 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : December 2013
Actual Primary Completion Date : August 2016
Actual Study Completion Date : November 2016


Arm Intervention/treatment
Experimental: Pacritinib, Once Daily
Pacritinib 400 mg taken orally, once daily
Drug: Pacritinib
Experimental: Pacritinib, Twice Daily
Pacritinib 200 mg taken orally, twice daily
Drug: Pacritinib
Active Comparator: Best Available Therapy
Best Available Therapy includes any physician-selected treatment for primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis, such as approved JAK2 inhibitors, and may include any treatment received before study entry. Best Available Therapy may include ruxolitinib, other approved JAK2 inhibitors, hydroxyurea, glucocorticoids, erythropoietic agents, immunomodulatory agents, mercaptopurine, danazol, interferons, cytarabine, melphalan, or other agents and may also include no treatment and symptom-directed treatment without myelofibrosis-specific treatment.
Drug: Best Available Therapy



Primary Outcome Measures :
  1. To compare the efficacy of two dose-schedule arms of pacritinib (pooled once-daily and twice-daily dosing arms) with that of Best Available Therapy [ Time Frame: Baseline to Week 24 ]
    To compare the efficacy of two dose-schedule arms of pacritinib (pooled once-daily and twice-daily dosing arms) with that of Best Available Therapy in patients with thrombocytopenia and primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis; the efficacy measure for this analysis is the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline to week 24 by magnetic resonance imaging (MRI) or computed tomography (CT) and the proportion of patients achieving a ≥ 50% reduction in total symptom score from baseline to Week 24 as measured by the Myeloproliferative Neoplasm Symptom Assessment Form 2.0.


Secondary Outcome Measures :
  1. To compare the efficacy of once-daily pacritinib with that of Best Available Therapy [ Time Frame: Baseline to Week 24 ]
    To compare the efficacy of once-daily pacritinib with that of Best Available Therapy, as assessed by the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline to Week 24 by magnetic resonance imaging (MRI) or computed tomography (CT) and the 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.

  2. To compare the efficacy of twice-daily pacritinib with that of Best Available Therapy [ Time Frame: Baseline to Week 24 ]
    To compare the efficacy of twice-daily pacritinib with that of Best Available Therapy, as assessed by the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline to Week 24 by magnetic resonance imaging (MRI) or computed tomography (CT) and the proportion of patients achieving a ≥ 50% reduction in the total symptom score from baseline to Week 24 on the myeloproliferate Neoplasm Symptom Assessment Form 2.0.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02055781


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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: CTI BioPharma
ClinicalTrials.gov Identifier: NCT02055781     History of Changes
Other Study ID Numbers: PERSIST-2 (PAC326)
First Posted: February 5, 2014    Key Record Dates
Last Update Posted: October 22, 2018
Last Verified: October 2018
Keywords provided by CTI BioPharma:
Myelofibrosis
Post-Polycythemia Vera Myelofibrosis
Post-Essential Thrombocythemia Myelofibrosis
Primary Myelofibrosis
Polycythemia
Polycythemia Vera
Thrombocythemia, Essential
Thrombocythemia
Myeloproliferative Disorders
Bone Marrow Disease
Hematologic Diseases
Blood Platelet Disorders
Hemorrhagic Disorders
Splenomegaly
Pacritinib
MPN-SAF
MPN-SAF TSS
Anemia
Myeloproliferative
Myeloproliferative Neoplasm
Spleen
Spleen volume
Thrombocytopenia
SB1518
Additional relevant MeSH terms:
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Polycythemia Vera
Thrombocytopenia
Primary Myelofibrosis
Polycythemia
Thrombocytosis
Thrombocythemia, Essential
Blood Platelet Disorders
Hematologic Diseases
Myeloproliferative Disorders
Bone Marrow Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Neoplasms
Blood Coagulation Disorders
Hemorrhagic Disorders