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Search / Trial NCT03165734

A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis

Launched by SWEDISH ORPHAN BIOVITRUM · May 23, 2017

Trial Information

Current as of July 23, 2025

Recruiting

Keywords

Myelofibrosis Pacritinib Post Polycythemia Vera Myelofibrosis Post Essential Thrombocythemia Myelofibrosis Ruxolitinib Bone Marrow Disease Hematologic Diseases Blood Platelet Disorders Hemorrhagic Disorders Splenomegaly Anemia Spleen Volume Spleen

ClinConnect Summary

This clinical trial is studying a medication called pacritinib to see how well it works for patients with specific types of myelofibrosis, which is a serious blood disorder that affects bone marrow. The trial is looking for adult patients who have low platelet counts (less than 50,000/μL) and notable symptoms related to their condition. About 399 participants will be enrolled, with two-thirds receiving pacritinib and one-third receiving other treatments chosen by their doctors.

To be eligible for the trial, participants must be at least 18 years old, have a confirmed diagnosis of myelofibrosis, and meet certain health criteria, such as having a specific level of platelet count and showing significant symptoms like pain or discomfort. Those who qualify will take the medication for a set period and will have regular check-ups to monitor their health and response to treatment. It's also important to know that participants will need to consent to the study and may be required to undergo tests like MRIs or CT scans during the trial. This study is currently recruiting participants, and it aims to provide new insights into treating myelofibrosis effectively.

Gender

ALL

Eligibility criteria

  • Diagnosis and Inclusion Criteria
  • 1. Primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF (as defined by Tefferi and Vardiman 2008
  • 2. Platelet count of \<50,000/μL at Screening (Day -35 to Day -3)
  • 3. Dynamic International Prognostic Scoring System Intermediate-1, Intermediate-2, or High-Risk (Passamonti et al 2010
  • 4. Palpable splenomegaly ≥5 cm below the lower costal margin (LCM) in the midclavicular line as assessed by physical examination
  • 5. TSS of ≥10 on the MPN-SAF TSS 2.0 or a single symptom score of ≥5 or two symptoms of ≥3, including only the symptoms of left upper quadrant pain, bone pain, itching, or night sweats.The TSS criteria need only to be met on a single day.
  • 6. Age ≥18 years
  • 7. Eastern Cooperative Oncology Group performance status 0 to 2
  • 8. Peripheral blast count of \<10% throughout the Screening period prior to randomization
  • 9. Absolute neutrophil count of ≥500/µL
  • 10. Left ventricular cardiac ejection fraction of ≥50% by echocardiogram or multigated acquisition scan
  • 11. Adequate liver and renal function, defined by liver transaminases (aspartate aminotransferase \[AST\]/serum glutamic-oxaloacetic transaminase \[SGOT\] and alanine aminotransferase \[ALT\]/serum glutamic pyruvic transaminase \[SGPT\]) ≤3 × the upper limit of normal (ULN) (AST/ALT ≤5 × ULN if transaminase elevation is related to MF), total bilirubin ≤4 x ULN (in cases where total bilirubin is elevated, direct bilirubin ≤4 × ULN, is required) and creatinine ≤2.5 mg/dL
  • 12. Adequate coagulation defined by prothrombin time/international normalized ratio and partial thromboplastin time ≤1.5 × ULN
  • 13. If fertile, willing to use highly effective birth control methods during the study
  • 14. Willing to undergo and able to tolerate frequent MRI or CT scan assessments during the study
  • 15. Able to understand and willing to complete symptom assessments using a patient-reported outcome instrument
  • 16. Provision of signed informed consent
  • Exclusion Criteria
  • 1. Life expectancy \<6 months
  • 2. Completed allogeneic stem cell transplant (allo-SCT) or are eligible for and willing to complete other approved available therapy including allogeneic stem cell
  • 3. History of splenectomy or planning to undergo splenectomy
  • 4. Splenic irradiation within the last 6 months
  • 5. Previously treated with pacritinib
  • 6. Treatment with any MF-directed therapy within 14 days prior to treatment Day 1
  • 7. Prior treatment with more than one JAK2 inhibitor
  • 8. Prior treatment with with ruxolitinib, if BOTH of the following conditions are met:
  • i. exposure to higher-dose ruxolitinib (\>10 mg daily) within 120 days prior to treatment Day 1 AND ii. total duration of treatment with higher-dose ruxolitinib (\>10 mg daily) was \>90 days, from first to last exposure (i.e., this 90-day period starts on the date of first administration of ruxolitinib at a total daily dose of \>10 mg and continues for 90 calendar days, regardless of whether higher-dose ruxolitinib is administered continuously or intermittently).
  • 9. Prior treatment with any JAK2 inhibitor other than ruxolitinib, irrespective of dose, with a duration of \>90 days. The 90-day period starts on the date of first administration of JAK2 inhibitor therapy and continues for 90 calendar days, regardless of whether therapy is administered continuously or intermittently.
  • 10. Treatment with an experimental therapy, including MF-directed experimental therapies within 28 days prior to treatment Day 1
  • 11. Systemic treatment with a strong CYP 3A4 inhibitor or a strong CYP 3A4 inducer within 14 days prior to treatment Day 1. Shorter washout periods may be permitted with approval of the Medical Monitor, provided that the washout period is at least five half-lives of the drug prior to treatment Day 1
  • 12. Significant recent bleeding history defined as National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) grade ≥2 within 3 months prior to treatment Day 1, unless precipitated by an inciting event (eg, surgery, trauma, or injury)
  • 13. Systemic treatment with medications that increase the risk of bleeding, including anticoagulants, antiplatelet agents (except for aspirin dosages of ≤100 mg per day),and daily use of cyclooxygenase-1 (COX-1) inhibiting non-steroidal anti-inflammatory drugs (NSAIDs) within 14 days prior to treatment Day 1. Treatment with systemic anti-vascular endothelial growth factor (anti-VEGF) agents within 28 days prior to treatment Day 1.
  • 14. Systemic treatment with medications that can prolong the QT interval within 14 days prior to treatment Day 1. Shorter washout periods may be permitted with approval of the Medical Monitor, provided that the washout period is at least five half-lives of the drug prior to treatment Day 1
  • 15. Any history of CTCAE grade ≥2 non-dysrhythmia cardiac conditions within 6 months prior to treatment Day 1. Patients with asymptomatic grade 2 non-dysrhythmia cardiovascular conditions may be considered for inclusion, with the approval of the Medical Monitor, if stable and unlikely to affect patient safety.
  • 16. Any history of CTCAE grade ≥2 cardiac dysrhythmias within 6 months prior to treatment Day 1. Patients with non-corrected QT interval CTCAE grade 2 cardiac dysrhythmias may be considered for inclusion, with the approval of the Medical Monitor, if the dysrhythmias are stable, asymptomatic, and unlikely to affect patient safety.
  • 17. QT corrected by the Fridericia method (QTcF) prolongation \>450 ms or other factors that increase the risk for QT interval prolongation (eg, hypokalemia \[defined as serum potassium \<3.0 mEq/L that is persistent and refractory to correction\], or history of long QT interval syndrome).
  • 18. New York Heart Association Class II, III, or IV congestive heart failure
  • 19. Any active gastrointestinal or metabolic condition that could interfere with absorption of oral medication
  • 20. Active or uncontrolled inflammatory or chronic functional bowel disorder such as Crohn's Disease, inflammatory bowel disease, chronic diarrhea, or chronic constipation
  • 21. Other malignancy within 3 years prior to treatment Day 1. The following patients may be eligible despite having had a malignancy within the prior 3 years: patients with curatively treated squamous or basal cell carcinoma of the skin; patients with curatively treated non-invasive cancers; patients with organ-confined prostate cancer with prostate specific antigen (PSA) \<20 ng/mL and National Comprehensive Cancer Network risk of Very Low, Low, or Favorable Intermediate; and patients with curatively treated non-metastatic prostate cancer with negative PSA.
  • 22. Uncontrolled intercurrent illness, including, but not limited to, ongoing active infection, psychiatric illness, or social situation that, in the judgment of the treating physician, would limit compliance with study requirements
  • 23. Known seropositivity for human immunodeficiency (HIV) virus. For patients in France, Czech Republic, and Italy only: testing for HIV is required during Screening.
  • 24. Known active hepatitis A, B, or C virus infection. For patients in France, Czech Republic and Italy only: testing for hepatitis B and C is required during Screening.
  • 25. Women who are pregnant or lactating
  • 26. Concurrent enrollment in another interventional trial
  • 27. Severe thrombocytopenia due to vitamin B12 deficiency, folate deficiency, or viral infection in the opinion of the investigator
  • 28. Known hypersensitivity to pacritinib or any of the following inactive ingredients: microcrystalline cellulose, polyethylene glycol, and magnesium stearate; any contraindication to the "physician's choice" medicinal product selected by the investigator to be used as the comparator or to loperamide or equivalent antidiarrheal medication
  • 29. Persons deprived of their liberty by a judicial or administrative decision
  • 30. Persons subject to legal protection measures or unable to express their consent
  • 31. Temporarily incapacitated persons

About Swedish Orphan Biovitrum

Swedish Orphan Biovitrum (Sobi) is a leading global biopharmaceutical company dedicated to developing and delivering innovative therapies for rare diseases and complex conditions. With a strong focus on hematology, immunology, and genetic disorders, Sobi leverages advanced research and development capabilities to provide life-changing treatments for patients in need. The company is committed to sustainability and collaboration, working closely with healthcare professionals, patient organizations, and regulatory authorities to enhance patient outcomes and improve quality of life. Sobi's extensive portfolio includes both proprietary and partnered products, reflecting its dedication to addressing unmet medical needs in the rare disease community.

Locations

New York, New York, United States

Baltimore, Maryland, United States

Cleveland, Ohio, United States

Seattle, Washington, United States

Chicago, Illinois, United States

Hackensack, New Jersey, United States

Chicago, Illinois, United States

Grand Rapids, Michigan, United States

Duarte, California, United States

Rochester, New York, United States

Columbus, Ohio, United States

Cleveland, Ohio, United States

Portland, Oregon, United States

Edmonton, Alberta, Canada

Weston, Florida, United States

Pittsburgh, Pennsylvania, United States

New York, New York, United States

Aurora, Colorado, United States

Washington, District Of Columbia, United States

New Haven, Connecticut, United States

Phoenix, Arizona, United States

Los Angeles, California, United States

Baltimore, Maryland, United States

Bucharest, , Romania

Houston, Texas, United States

Madrid, , Spain

Durham, North Carolina, United States

Seoul, , Korea, Republic Of

Seoul, , Korea, Republic Of

New York, New York, United States

Sydney, New South Wales, Australia

Chicago, Illinois, United States

New Orleans, Louisiana, United States

Seoul, , Korea, Republic Of

Busan, , Korea, Republic Of

Daegu, , Korea, Republic Of

Brno, , Czechia

Seoul, , Korea, Republic Of

Westwood, Kansas, United States

Columbus, Ohio, United States

Los Angeles, California, United States

Fukushima, , Japan

Miyazaki, , Japan

Tokyo, , Japan

Tokyo, , Japan

Tokyo, , Japan

Bucharest, , Romania

Toronto, Ontario, Canada

Kraków, , Poland

Paris, , France

Madrid, , Spain

Pamplona, , Spain

Washington, District Of Columbia, United States

Salt Lake City, Utah, United States

Fukuoka, , Japan

Manchester, , United Kingdom

Tsu, , Japan

Florence, , Italy

Oxford, , United Kingdom

Kaposvár, , Hungary

Busan, , Korea, Republic Of

Olomouc, , Czechia

Gloucester, , United Kingdom

Toronto, Ontario, Canada

Miyagi, , Japan

Sofia, , Bulgaria

Varna, , Bulgaria

Seoul, , Korea, Republic Of

Gomel, , Belarus

Porto Alegre, Rio Grande Do Sul, Brazil

Saint John's, Newfoundland And Labrador, Canada

Barcelona, , Spain

Plovdiv, , Bulgaria

Volgograd, , Russian Federation

Grodno, , Belarus

Novi Sad, , Serbia

Salvador, Bahia, Brazil

Banja Luka, , Bosnia And Herzegovina

Bethesda, Maryland, United States

Saint Louis, Missouri, United States

Strasbourg, , France

Pessac, , France

Barcelona, , Spain

San Antonio, Texas, United States

Monza, , Italy

Edmonton, Alberta, Canada

Birmingham, Alabama, United States

Boulder, Colorado, United States

Bethesda, Maryland, United States

Columbia, Maryland, United States

Boston, Massachusetts, United States

Ann Arbor, Michigan, United States

Las Vegas, Nevada, United States

Commack, New York, United States

New York, New York, United States

Nashville, Tennessee, United States

San Antonio, Texas, United States

Melbourne, Victoria, Australia

Perth, Western Australia, Australia

Minsk, , Belarus

Pleven, , Bulgaria

Sofia, , Bulgaria

Calgary, Alberta, Canada

Vancouver, British Columbia, Canada

Halifax, Nova Scotia, Canada

Montreal, Quebec, Canada

Pilsen, , Czechia

Prague, , Czechia

Amiens, , France

Nîmes, , France

Pierre Benite, , France

Poitiers, , France

Toulouse, , France

Tbilisi, , Georgia

Tbilisi, , Georgia

Tbilisi, , Georgia

Tbilisi, , Georgia

Cologne, , Germany

Halle, , Germany

Minden, , Germany

Munich, , Germany

Ulm, , Germany

Budapest, , Hungary

Debrecen, , Hungary

Kecskemét, , Hungary

Nyíregyháza, , Hungary

Székesfehérvár, , Hungary

Haifa, , Israel

Jerusalem, , Israel

Kfar Saba, , Israel

Petah Tikva, , Israel

Tel Aviv, , Israel

Bari, , Italy

Bologna, , Italy

Forlì, , Italy

Palermo, , Italy

Pavia, , Italy

Rimini, , Italy

Rome, , Italy

Varese, , Italy

Gdańsk, , Poland

Rzeszów, , Poland

Toruń, , Poland

Warsaw, , Poland

Wrocław, , Poland

łódź, , Poland

Moscow, , Russian Federation

Moscow, , Russian Federation

Moscow, , Russian Federation

Pyatigorsk, , Russian Federation

Saint Petersburg, , Russian Federation

Saint Petersburg, , Russian Federation

Saint Petersburg, , Russian Federation

Samara, , Russian Federation

Ufa, , Russian Federation

Belgrade, , Serbia

Barcelona,, , Spain

Murcia, , Spain

Salamanca, , Spain

Valencia, , Spain

Cherkasy, , Ukraine

Dnipro, , Ukraine

Ivano Frankivs'k, , Ukraine

Kharkiv, , Ukraine

Kyiv, , Ukraine

Kyiv, , Ukraine

Kyiv, , Ukraine

Lviv, , Ukraine

Poltava, , Ukraine

Sheffield, South Yorkshire, United Kingdom

Glasgow, , United Kingdom

London, , United Kingdom

London, , United Kingdom

London, , United Kingdom

Seville, , Spain

Lublin, , Poland

Tōon, , Japan

Bengaluru, , India

Turin, , Italy

Tbilisi, , Georgia

Chandigarh, , India

Udine, , Italy

Saint John's, Newfoundland And Labrador, Canada

Brescia, , Italy

Tbilisi, , Georgia

Katowice, , Poland

Braşov, , Romania

Cluj Napoca, , Romania

Nowy Sącz, , Poland

Marseille, , France

Milan, , Italy

Katowice, , Poland

Novara, , Italy

Sarajevo, , Bosnia And Herzegovina

Naples, , Italy

Madrid, , Spain

Białystok, , Poland

Rome, , Italy

Pune, Maharashtra, India

Calgary, Alberta, Canada

Vancouver, British Columbia, Canada

Halifax, Nova Scotia, Canada

Montreal, Quebec, Canada

Guwahati, Assam, India

Jaipur, Rajasthan, India

Kolkata, West Bengal, India

Aktobe, , Kazakhstan

Almaty, , Kazakhstan

Karaganda, , Kazakhstan

Shymkent, , Kazakhstan

Fortaleza, Ceara, Brazil

Goiana, Goias, Brazil

Belo Horizonte, Minas Gerais, Brazil

Curitiba, Parana, Brazil

Mossoró, Rio Grande Do Norte, Brazil

Porto Alegre, Rio Grande Do Sul, Brazil

Florianópolis, Santa Catarina, Brazil

Campinas, Sao Paulo, Brazil

Jaú, Sao Paulo, Brazil

São Paulo, Sao Paulo, Brazil

São Paulo, Sao Paulo, Brazil

São Paulo, Sao Paulo, Brazil

Rio De Janeiro, , Brazil

Sūrat, Gujarat, India

Gurgaon, Haryana, India

Madurai, Tamil Nadu, India

Hyderabad, Telangana, India

Dehradun, Uttarakhand, India

Kolkata, West Bengal, India

Delhi, , India

Chuo, , Japan

Astana, , Kazakhstan

Astana, , Kazakhstan

Ust Kamenogorsk, , Kazakhstan

Patients applied

0 patients applied

Trial Officials

Simran Singh

Study Director

Sobi, Inc.

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

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