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A Clinical Study of Ruxolitinib in Patients With Primary Myelofibrosis (PM), Post-polycythemia Vera (PV) Myelofibrosis, or Post-essential Thrombocythemia (ET) Myelofibrosis

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ClinicalTrials.gov Identifier: NCT02087059
Recruitment Status : Completed
First Posted : March 14, 2014
Results First Posted : July 11, 2016
Last Update Posted : July 11, 2016
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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Brief Summary:
This is an open-label, multicenter clinical study in order to collect and examine data concerning the safety and efficacy of ruxolitinib in patients with Primary Myelofibrosis (MF), Post-Polycythemia Vera (PV) MF, Post-Essential Thrombocythemia (ET) MF.

Condition or disease Intervention/treatment Phase
Primary Myelofibrosis (MF) Drug: Ruxolitinib Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 51 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-label Clinical Study of the JAK Inhibitor Ruxolitinib (INC424) in Patients With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis
Study Start Date : April 2014
Actual Primary Completion Date : March 2015
Actual Study Completion Date : April 2015

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Ruxolitinib
Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.
 Drug: Ruxolitinib


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 24 weeks ]

Secondary Outcome Measures :
  1. Charge in Spleen Size From Baseline at Specified Week [ Time Frame: Baseline, 24 weeks ]
    Number of patients with spleen length reduced by ≥ 50% at specified week

  2. Charge in Spleen Size From Baseline up to the Specified Week [ Time Frame: Baseline, 24 weeks ]
    Number of patients with spleen length reduced by ≥ 50% up to specified week

  3. Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time [ Time Frame: 24 weeks ]
    The modified MFSAF v2.0 diary captures a patient's symptom severity on a scale of 0 (absent) to 10 (worst imaginable),with a maximal summary score of 60

  4. Summary of Summary of EORTC QLQ-C30 Responses by Time [ Time Frame: 24 weeks ]
    The QLQ-C30 version 1.0 (QLQ-C30) incorporates five functional scales (physical, role, cognitive, emotional, and social), three symptom scales (fatigue, pain, and nausea and vomiting), a global health status / QoL scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of the disease.All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Thus a high score for a functional scale represents a high / healthy level of functioning, a high score for the global health status / QoL represents a high QoL, but a high score for a symptom scale / item represents a high level of symptomatology / problems.


Eligibility Criteria
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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:

  1. ≥18 years of age
  2. Diagnosis of PMF, PPV-MF, or PET-MF, regardless of JAK2 mutational status. The diagnostic of PMF will be according to the World Health Organization (WHO) criteria (Thiele et al., 2008) and PPV-MF and PET-MF according to the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria (Barosi et al., 2008).
  3. At least one risk factors provided in the definition of IWG-MRT (Cervantes et al., 2009; classified as intermediate risk-1, intermediate risk-2, or high risk)
  4. Patients with intermediate risk-1 (patients who have only one of the IMG-MRT risk factors indicated above ) must have palpable splenomegaly with a length of ≥5 cm from the costal margin to the point of the greatest spleen protrusion.
  5. Proportion of blasts in peripheral blood <10%
  6. ECOG performance status of 0 to 2

  7. The following values for bone marrow function prior to treatment:

    1. Absolute neutrophil count ≥1,000/μL, and
    2. Platelet count ≥50,000/μL without administration of a growth factor, thrombopoietin, or platelet transfusion
  8. Stem cell transplantation is not a treatment option at present because it is not indicated or because there are no suitable donors.
  9. All drugs used to treat MF were discontinued at least 28 days before treatment initiation.
  10. Informed consent form should be signed before any screening procedures is performed

Exclusion Criteria:

  1. Hepatic or renal impairment as indicated by the following:

    • Direct bilirubin ≥2-fold than the upper limit of normal (ULN)
    • Alanine aminotransferase (ALT) >2.5-fold ULN
    • Creatinine >2.0 mg/dL
  2. Clinically significant infection by bacteria, fungus, mycobacteria, parasite, or virus (screening and enrollment postponed until completion of antibiotic treatment in patients with an acute bacterial infection that requires antibiotic use)
  3. Active hepatitis A, B, or C or HIV infection defined by a positive IgM-HA Ab test [hepatitis A virus antibody (immunoglobulin M [IgM])], HBs Ag test (hepatitis B surface antigen), HCV Ab test (hepatitis C virus antibody), or HIV Ab (human immunodeficiency virus antibody) at screening.
  4. History of malignancy within the previous 3 years, except for early-stage squamous cell carcinoma and basal cell carcinoma.
  5. History of serious congenital or acquired hemorrhagic disease
  6. Previous platelet count <25,000/μL or absolute neutrophil count <500/μL, except for patients currently undergoing treatment for a myeloproliferative neoplasm or cytotoxic therapy for any other reason.
  7. Splenic irradiation within 12 months before screening
  8. Administration of hematopoietic growth factor receptor agonists (erythropoietin, granulocyte colony stimulating factor, romiplostim, eltrombopag) within 14 days before screening or 28 days before treatment initiation.
  9. Currently receiving another investigational drug, or received another investigational drug within 30 days before the start of treatment.
  10. History of myocardial infarction or acute coronary syndrome within 6 months before screening
  11. Poorly controlled or unstable angina at present
  12. Rapid or paroxysmal atrial fibrillation at present
  13. Active alcohol or drug addiction that could hinder the patient's ability to comply with the study's requirements
  14. Pregnant or currently breastfeeding woman
  15. Women of childbearing potential or men with reproductive ability who are unwilling to take appropriate contraception measures
  16. Patient with any concurrent condition that, in the Investigator's opinion, would jeopardize the safety of the patient or compliance with the protocol
  17. History of hypersensitivity to the study drug or a drug with a similar chemical structure
Contacts and Locations
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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): NCT02087059


Locations
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Japan
Novartis Investigative Site
Nagoya-city, Aichi, Japan, 467-8602
Novartis Investigative Site
Matsuyama, Ehime, Japan, 790-8524
Novartis Investigative Site
Toon-city, Ehime, Japan, 791-0295
Novartis Investigative Site
Fukuoka-city, Fukuoka, Japan, 812-8582
Novartis Investigative Site
Kurume-city, Fukuoka, Japan, 830-0011
Novartis Investigative Site
Maebashi-city, Gunma, Japan, 371-8511
Novartis Investigative Site
Sapporo-city, Hokkaido, Japan, 060-8543
Novartis Investigative Site
Sapporo-city, Hokkaido, Japan, 060-8648
Novartis Investigative Site
Kobe-city, Hyogo, Japan, 650-0017
Novartis Investigative Site
Kobe-city, Hyogo, Japan, 650-0047
Novartis Investigative Site
Kumamoto City, Kumamoto, Japan, 860-8556
Novartis Investigative Site
Kyoto-city, Kyoto, Japan, 606-8507
Novartis Investigative Site
Tsu-city, Mie, Japan, 514-8507
Novartis Investigative Site
Sendai-city, Miyagi, Japan, 980-8574
Novartis Investigative Site
Miyazaki-city, Miyazaki, Japan, 889-1692
Novartis Investigative Site
Okayama-city, Okayama, Japan, 700-8558
Novartis Investigative Site
Hirakata-city, Osaka, Japan, 573-1191
Novartis Investigative Site
OsakaSayama, Osaka, Japan, 589-8511
Novartis Investigative Site
Suita-city, Osaka, Japan, 565-0871
Novartis Investigative Site
Shimotsuke-city, Tochigi, Japan, 329-0498
Novartis Investigative Site
Bunkyo-ku, Tokyo, Japan, 113-8431
Novartis Investigative Site
Bunkyo-ku, Tokyo, Japan, 113-8519
Novartis Investigative Site
Bunkyo-ku, Tokyo, Japan, 113-8603
Novartis Investigative Site
Bunkyo-ku, Tokyo, Japan, 113-8677
Novartis Investigative Site
Shinjuku-ku, Tokyo, Japan, 160-0023
Novartis Investigative Site
Shinjuku-ku, Tokyo, Japan, 162-8666
Novartis Investigative Site
Chuo-city, Yamanashi, Japan, 409-3898
Novartis Investigative Site
Akita, Japan, 010-8543
Novartis Investigative Site
Gifu, Japan, 501-1194
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02087059    
Other Study ID Numbers: CINC424AJP01
First Posted: March 14, 2014    Key Record Dates
Results First Posted: July 11, 2016
Last Update Posted: July 11, 2016
Last Verified: May 2016
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Post-Polycythemia Vera (PV) MF
Post-Essential Thrombocythemia (ET) MF
Additional relevant MeSH terms:
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Polycythemia Vera
Primary Myelofibrosis
Polycythemia
Thrombocytosis
Thrombocythemia, Essential
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
 Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Neoplasms
Blood Platelet Disorders
Blood Coagulation Disorders
Hemorrhagic Disorders