Azacitidine in Treating Patients With Myelofibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00381693
Recruitment Status : Terminated (Due to lack of accrual and trial has demonstrated too little clinical benefit)
First Posted : September 28, 2006
Results First Posted : January 24, 2011
Last Update Posted : April 21, 2011
National Cancer Institute (NCI)
Information provided by:
Mayo Clinic

September 26, 2006
September 28, 2006
November 9, 2010
January 24, 2011
April 21, 2011
August 2006
March 2008   (Final data collection date for primary outcome measure)
Patients With Confirmed Response (Complete Remission or Partial Remission on 2 Consecutive Evaluation at Least 4 Weeks Apart) During the First 4 Months of Treatment [ Time Frame: 4 months ]

Response Definitions:

  • Completion Remission (CR):complete resolution of disease-related symptoms, ultrasound-documented resolution of hepastosplenomegaly, normalization of the peripheral blood count, white cell differential, and smear, normalization of bone marrow histology including disappearance of fibrosis and osteosclerosis. Residual cytogenetic abnormalities are allowed.
  • Partial Remission (PR): a major response in any baseline applicable criteria (except constitutional symptoms) without progression in any other category.
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Complete list of historical versions of study NCT00381693 on Archive Site
  • Overall Survival (OS) [ Time Frame: From date of registration until death or 3 years after registration if patient is still alive ]
    OS was defined as the time from registration to death due to any cause or time from registration to 3 years after registration if patient is still alive.
  • Time to Progression [ Time Frame: up to 3 years ]
    Time to progression was defined as the time from registration to progression of disease. Those who die without documentation of disease progression will be considered to have had disease progression at the time of their death unless documented evidence clearly indicates no progression has occured.
  • Number of Participants With Treatment Related Adverse Events [ Time Frame: Every 4 weeks during treatment ]

    Adverse events (AE) that are classified as either possibly, probably, or definitely related to study treatment according to the National Cancer Institute Common Toxicity Criteria for Adverse Events (NCI CTCAE version 3.0). The maximum grade for each type of AE will be recorded for each patient. Grade refers to the severity of the AE.

    Grade 1: Mild AE, Grade 2: Moderate AE, Grade 3: Severe AE, Grade 4: Life-threatening or disabling AE, Grade 5: Death related AE

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Azacitidine in Treating Patients With Myelofibrosis
Phase II Study of Azacitidine in Myelofibrosis

RATIONALE: Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying how well azacitidine works in treating patients with myelofibrosis.



  • Determine the efficacy of azacitidine in patients with myelofibrosis (MF) with myeloid metaplasia.
  • Evaluate the safety of azacitidine in these patients. Secondary
  • Evaluate pertinent biologic characteristics of MF before and during therapy with azacitidine.
  • Assess the effects of study treatment on constitutional symptoms in these patients.
  • Estimate time to event distributions for overall survival and progression. OUTLINE: Patients receive azacitidine subcutaneously once daily on days 1-5. Treatment repeats every 4 weeks for up to 12 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed periodically for up to 3 years.

PROJECTED ACCRUAL: A total of 35 patients will be accrued for this study.

Phase 2
Masking: None (Open Label)
Primary Purpose: Treatment
  • Chronic Myeloproliferative Disorders
  • Secondary Myelofibrosis
Drug: azacitidine
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
April 2009
March 2008   (Final data collection date for primary outcome measure)


  • Histologically confirmed myelofibrosis with myeloid metaplasia (MMM), including any of the following subtypes:

    • Agnogenic myeloid metaplasia
    • Post-polycythemic myeloid metaplasia
    • Post-thrombocythemic myeloid metaplasia
  • Evaluable and symptomatic disease, defined as 1 of the following:

    • Anemia (hemoglobin < 10 g/dL or erythrocyte transfusion-dependent, requiring 1 transfusion ≤ 8 weeks)
    • Treatment required* for symptomatic palpable splenomegaly (palpable hepatomegaly is acceptable if previously splenectomized) NOTE: *Subjective but painful enough to mandate intervention
  • Absence of t(9;22) by fluorescent in situ hybridization (FISH) or standard cytogenetics (by peripheral blood or marrow)

    - Previous demonstration of a lack of this translocation (at any point) is sufficient

  • No advanced malignant hepatic tumors


  • ECOG performance status 0-2
  • Absolute neutrophil count ≥ 1,000/mm³
  • Platelet count ≥ 50,000/mm³
  • Creatinine ≤ 2.0 mg/dL
  • Total bilirubin ≤ 2.0 mg/dL OR direct bilirubin ≤ 2.0 mg/dL if total bilirubin elevated (unless attributed to underlying disease)
  • AST and ALT ≤ 2 times upper limit of normal (unless clinically attributed to hepatic extramedullary hematopoiesis)
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No baseline peripheral or autonomic neuropathy ≥ grade 2
  • No condition, including the presence of laboratory abnormalities, that would preclude study compliance
  • No hypersensitivity to mannitol or azacitidine
  • Not incarcerated in a municipality (i.e., county, state, or federal prison)


  • At least 14 days since prior chemotherapy, including interferon alfa, anagrelide, or other myelosuppressive agents
  • At least 14 days since prior systemic corticosteroids
  • At least 14 days since prior investigational agents
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
United States
P30CA015083 ( U.S. NIH Grant/Contract )
MC058D ( Other Identifier: Mayo Clinic Cancer Center )
05-004297 ( Other Identifier: Mayo Clinic IRB )
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Ruben A. Mesa, Mayo Clinic Cancer Center
Mayo Clinic
National Cancer Institute (NCI)
Study Chair: Ruben A. Mesa, MD Mayo Clinic
Mayo Clinic
April 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP