Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia
- Conditions
- Post-polycythemia Vera MyelofibrosisPrimary MyelofibrosisPost-essential Thrombocythemia Myelofibrosis
- Interventions
- Drug: Best Available TherapyDrug: Pacritinib
- Registration Number
- NCT02055781
- Lead Sponsor
- CTI BioPharma
- 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.
- 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 311
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Best Available Therapy 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. Pacritinib, Once Daily Pacritinib Pacritinib 400 mg QD Pacritinib, Twice Daily Pacritinib Pacritinib 200 mg BID
- Primary Outcome Measures
Name Time Method Spleen Volume Reduction 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).
Total Symptom Score (TSS) Reduction 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.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (122)
SCRI- Florida Cancer Specialists South Region
🇺🇸Fort Myers, Florida, United States
Saint John Regional Hospital
🇨🇦Saint John, New Brunswick, Canada
Princess Margaret Cancer Center
🇨🇦Toronto, Ontario, Canada
Nebraska Hematology-Oncology, P.C.
🇺🇸Lincoln, Nebraska, United States
Froedtert Hospital and the Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Hematology-Oncology Associates of Northern Jersey
🇺🇸Morristown, New Jersey, United States
Providence Cancer Institute
🇺🇸Southfield, Michigan, United States
Centre Hospitalier de Jolimont-Lobbes
🇧🇪Haine-Saint-Paul, Hainaut, Belgium
Royal Adelaide Hospital
🇦🇺Adelaide, South Australia, Australia
UC Louvain
🇧🇪Yvoir, Belgium
Chu d'Amiens Hopital Sud
🇫🇷Amiens, Cedex 1, France
Republican Hopsital n.a. V.A. Baranov
🇷🇺Petrozavodsk, Russian Federation
National Haematology Research Center
🇷🇺Moscow, Russian Federation
UPMC Hillman Cancer Center
🇺🇸Pittsburgh, Pennsylvania, United States
Houston Methodist
🇺🇸Houston, Texas, United States
Norton Cancer Institute, Suburban
🇺🇸Louisville, Kentucky, United States
University of Texas MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
Investigative Clinical Research of Indiana
🇺🇸Indianapolis, Indiana, United States
George Washington University- Medical Faculty Associates
🇺🇸Washington, District of Columbia, United States
SCRI - Florida Cancer Specialists North Region
🇺🇸Saint Petersburg, Florida, United States
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
H. Lee Moffitt Cancer Center & Research Institute
🇺🇸Tampa, Florida, United States
Siouxland Hematology-Oncology Associates, L.L.P (SHOA)
🇺🇸Sioux City, Iowa, United States
Stony Brook University Medical Center
🇺🇸Stony Brook, New York, United States
SCRI-Oncology Hematology Care
🇺🇸Cincinnati, Ohio, United States
Fred Hutchinson Cancer Research Center
🇺🇸Seattle, Washington, United States
Providence Regional Cancer Partnership
🇺🇸Everett, Washington, United States
University Hospital Ulm
🇩🇪Ulm, Germany
Green Bay Oncology
🇺🇸Green Bay, Wisconsin, United States
St George Hospital
🇦🇺Kogarah, New South Wales, Australia
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Monash Health - Monash Medical Centre
🇦🇺Clayton, Victoria, Australia
Prince of Wales Hospital
🇦🇺Randwick, Australia
ZNA - Stuivenberg
🇧🇪Antwerpen, Belgium
Hopital Brugmann
🇧🇪Brussels, Belgium
Fakultní nemocnice Brno
🇨🇿Brno, NAP, Czechia
Hôpital Caremeau
🇫🇷Nimes, Cedex 9, France
University Hospital Hradec Kralove
🇨🇿Králová, Czechia
Faculty Hospital Olomouc
🇨🇿Olomouc, NAP, Czechia
Fakultní nemocnice Plzeň
🇨🇿Plzeň, NAP, Czechia
CH de Mulhouse
🇫🇷Mulhouse, Cedex, France
CHU de CAEN
🇫🇷Caen, France
Saint Antoine Hospital
🇫🇷Paris, France
University Hospital Essen
🇩🇪Essen, Germany
Charite-Medical University
🇩🇪Berlin, Germany
Gemeinschaftspraxis Hämatologie/Onkologie
🇩🇪Dresden, Germany
Uniklinik Freiburg
🇩🇪Freiburg, Germany
Universitatsklinikum Halle (Saale)
🇩🇪Halle (Saale), Germany
Klinik I fur Innere Medizin, Universitat Koln
🇩🇪Koln, Germany
Städtisches Klinikum München GmbH
🇩🇪Munchen, Germany
University of Munster
🇩🇪Munster, Germany
Semmelweis Egyetem AOK
🇭🇺Budapest, Hungary
University of Debrecen, Belgyogyaszati Intezet
🇭🇺Debrecen, Hungary
Bekes Megyei Pandy Kalman Korhaz
🇭🇺Gyula, Hungary
SZTE II. sz Belgyogyoszati Klinika es Kardiologiai Kozpont
🇭🇺Szeged, Hungary
Kaposi Mór Oktató Kórház
🇭🇺Kaposvár, Hungary
Auckland District Health Board, Auckland City Hospital
🇳🇿Auckland, New Zealand
Jász-Nagykun-Szolnok Megyei Hetényi Géza Kórház-Rendelőint
🇭🇺Szolnok, Hungary
Erasmus MC
🇳🇱Rotterdam, Netherlands
University Hospital Maastricht
🇳🇱Maastricht, Netherlands
Middlemore Hospital
🇳🇿Auckland, New Zealand
North Shore Hospital
🇳🇿Takapuna, New Zealand
Canterbury District Health Board
🇳🇿Christchurch, New Zealand
CCDHB - Wellington Hospital
🇳🇿Wellington, New Zealand
Saratov State Medical University
🇷🇺Saratov, Saratov Region, Russian Federation
Bashkir State Medical University
🇷🇺Ufa, Republic Of Bashkortostan, Russian Federation
Ryazan Regional Clinical Hospital
🇷🇺Ryazan, Russian Federation
Russian Research Institute of Hematology and Transfusiology
🇷🇺St. Petersburg, Russian Federation
Military Medical Academy n.a. S.M. Kirov
🇷🇺St. Petersburg, Russian Federation
Beatson West of Scotland Cancer Centre
🇬🇧Glasgow, United Kingdom
Hammersmith Hosp - ICH NHS Trust
🇬🇧London, United Kingdom
Oxford University Hospitals NHS Trust
🇬🇧Oxford, United Kingdom
Royal Hallamshire Hospital
🇬🇧Sheffield, United Kingdom
Mayo Clinic Arizona
🇺🇸Scottsdale, Arizona, United States
Henry Ford Health System
🇺🇸Detroit, Michigan, United States
University of Oklahoma
🇺🇸Oklahoma City, Oklahoma, United States
Sarah Cannon Research Institute (SCRI)
🇺🇸Nashville, Tennessee, United States
Huntsman Cancer Hospital
🇺🇸Salt Lake City, Utah, United States
City of Hope
🇺🇸Duarte, California, United States
USC Norris Comprehensive Cancer Center
🇺🇸Los Angeles, California, United States
Moores Cancer Centre
🇺🇸La Jolla, California, United States
Carle Cancer Center
🇺🇸Urbana, Illinois, United States
UT Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Mount Sinai Medical Center
🇺🇸New York, New York, United States
UTMB Galveston
🇺🇸Galveston, Texas, United States
Hackensack University
🇺🇸Hackensack, New Jersey, United States
Washington University School of Medicine Division of Oncology
🇺🇸Saint Louis, Missouri, United States
Arizona Clinical Research Center
🇺🇸Tucson, Arizona, United States
Stanford Cancer Center
🇺🇸Stanford, California, United States
Indiana University Goshen Cancer Centre
🇺🇸Goshen, Indiana, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
Rocky Mountain Cancer Center
🇺🇸Boulder, Colorado, United States
St Joseph Mercy Hospital
🇺🇸Ann Arbor, Michigan, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Texas Onocolgy-Baylor Sammons Cancer Center
🇺🇸Dallas, Texas, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Cleveland Clinic-Taussig Cancer Center
🇺🇸Cleveland, Ohio, United States
Upstate Oncology Associates
🇺🇸Greenville, South Carolina, United States
Virginia Cancer Specialists
🇺🇸Leesburg, Virginia, United States
Cancer Care Centers of South Texas
🇺🇸San Antonio, Texas, United States
Box Hill Hospital
🇦🇺Box Hill, Victoria, Australia
Perth Blood Institute
🇦🇺Nedlands, Western Australia, Australia
Haematology and Oncology Clinics of Australia
🇦🇺Chermside, Australia
CHU Purpan
🇫🇷Toulouse, Cedex 9, France
Hopital l'Archet, CHU de Nice
🇫🇷Nice, France
AZ Sint Jan Brugge-Oostende AV
🇧🇪Brugge, Belgium
Cliniques Universitaires St-Luc
🇧🇪Bruxelles, Belgium
St Augustinus
🇧🇪Wilrijk, Belgium
University Hospital Leipzig
🇩🇪Leipzig, Germany
CHU Rennes
🇫🇷Rennes, Cedex 9, France
Centre Hospitalier de Lens
🇫🇷Lens, France
CHU de Strasbourg
🇫🇷Strasbourg, France
Centre Hospitalier Lyon Sud
🇫🇷Pierre Benite, France
Institut Gustave Roussy
🇫🇷Villejuif Cedex, France
Birmingham Heartlands Hospital
🇬🇧Birmingham, United Kingdom
The Christie NHS Foundation Trust
🇬🇧Manchester, United Kingdom
Leicester Royal Infirmary
🇬🇧Leicester, United Kingdom
New Mexico Cancer Care Alliance
🇺🇸Albuquerque, New Mexico, United States
Belfast Health and Social Care Trust
🇬🇧Belfast, N. Ireland, United Kingdom
Guy's Hospital
🇬🇧London, United Kingdom
Thomas Jefferson University
🇺🇸Philadelphia, Pennsylvania, United States
Royal Liverpool University Hospital
🇬🇧Liverpool, Merseyside, United Kingdom
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