Ruxolitinib Phosphate and Danazol in Treating Anemia in Patients with Myelofibrosis

Overview

  • Study type

    Interventional
  • Study phase

    II
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Scottsdale/Phoenix, Arizona: 12-002466
    NCT ID: NCT01732445
    Sponsor Protocol Number: MC1283

About this study

This phase II pilot trial studies how well ruxolitinib phosphate and danazol work in treating anemia in patients with myelofibrosis. Ruxolitinib phosphate and danazol may cause the body to make more red blood cells. They are used to treat anemia in patients with myelofibrosis.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Study closed to enrollment

Inclusion Criteria:

  • Histological confirmation of primary myelofibrosis (MF), post polycythemia vera (PV) or post essential thrombocythemia (ET) myelofibrosis (intermediate 1, intermediate II or high risk) requiring medical therapy
  • Anemia is required for trial entry (defined as hemoglobin < 10g/dL or transfusion dependent [having needed a transfusion anytime in the past 6 months])
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 at study entry
  • Absolute neutrophil count (ANC) ≥ 1000/uL
  • Platelet count ≥ 50,000/uL
  • Serum creatinine ≤ 1.5 x the upper limit of normal (ULN)
  • Total bilirubin ≤ 1.5 x ULN; if total bilirubin is > 1.5 x ULN, a direct bilirubin should be performed and must be < 1.5mg/dL
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 x ULN; higher values (i.e., ≤ 5 x ULN) are allowed if clinically compatible with hepatic extramedullary hematopoiesis
  • Life expectancy of ≥ 6 months
  • Patient able to provide voluntary written informed consent to participate
  • Willing to comply with scheduled visits, treatment plans, laboratory assessments, and other study-related procedures
  • Negative pregnancy test done ≤ 7 days prior to registration, for women of childbearing potential only

Exclusion Criteria:

  • Any chemotherapy (e.g., hydroxyurea), immunomodulatory drug therapy (e.g., thalidomide, interferon-alpha), immunosuppressive therapy, corticosteroids > 10 mg/day prednisone or equivalent, or growth factor treatment (e.g., erythropoietin), hormones (e.g., androgens, danazol) ≤ 14 days prior to registration; note: patients who are on ruxolitinib may continue on without a 14 day wash out at the treating physician's discretion
  • Major surgery ≤ 28 days or radiation ≤ 6 months prior to registration
  • Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
  • Active acute infection requiring antibiotics
  • Uncontrolled congestive heart failure (New York Heart Association classification 3 or 4), angina, myocardial infarction, cerebrovascular accident, coronary/peripheral artery bypass, graft surgery, transient ischemic attack, or pulmonary embolism within 3 months prior to registration
  • Participation in any study of an investigational agent (drug, biologic, device) ≤ 30 days, unless during non-treatment phase
  • Any of the following:
    • Pregnant women
    • Nursing women
    • Men or women of childbearing potential who are unwilling to employ adequate contraception
  • Known human immunodeficiency virus or acquired immunodeficiency syndrome-related illness
  • Clinically active hepatitis B or C
  • Active malignancy other than MF, except adequately treated basal cell carcinoma and squamous cell carcinoma of the skin, cervical carcinoma in situ or other malignancies that have been stable and off therapy for 5 years
  • Patient currently taking simvastatin, or lovastatin at a dose greater than 10 mg/day
  • Men with prostate specific antigen (PSA) > 4 ng/ml or with uncontrolled benign prostatic hypertrophy
  • Patient received prior combination treatment with ruxolitinib and danazol together; note: previous treatment with ruxolitinib and/or danazol as single agent therapy is allowed
  • Receiving any medications or substances that are strong or moderate inhibitors of cytochrome P450, family 3, subfamily A, polypeptide 4 (CYP3A4); use of the following strong or moderate inhibitors are prohibited ≤ 7 days prior to registration
    • Strong inhibitors of CYP3A4:
      • Indinavir (Crixivan)
      • Nelfinavir (Viracept)
      • Atazanavir (Reyataz)
      • Clarithromycin (Biaxin, Biaxin XL)
      • Itraconazole (Sporanox)
      • Ketoconazole (Nizoral)
      • Nefazodone (Serzone)
      • Saquinavir (Fortovase, Invirase)
      • Telithromycin (Ketek)
    • Moderate inhibitors of CYP3A4
      • Erythromycin (Erythrocin, E.E.S., Ery-Tab, Eryc, EryPed, PCE)
      • Fluconazole (Diflucan)
      • Grapefruit juice
      • Verapamil (Calan, Calan SR, Covera-HS, Isoptin SR, Verelan)
      • Verelan PM
      • Diltiazem (Cardizem, Cardizem CD, Cardizem LA, Cardizem SR, Cartia XT, Dilacor XR, Diltia XT, Taztia XT, Tiazac)

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Ruben Mesa, M.D.

Closed for enrollment

Contact information:

Research Information Center

800-664-4542

Additional contact information

Cancer-related trials contact form

Phone: 855-776-0015 (toll-free)

International patient clinical studies questions