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Imatinib Mesylate 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: NCT00245128
Recruitment Status : Terminated (Per PI, results from another similar study were published prior to study analysis. Negative study results were published therefore analysis was not completed)
First Posted : October 27, 2005
Results First Posted : January 13, 2012
Last Update Posted : January 13, 2012
National Cancer Institute (NCI)
Information provided by (Responsible Party):
OHSU Knight Cancer Institute

Brief Summary:

RATIONALE: Imatinib mesylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

PURPOSE: This phase II trial is studying the side effects of imatinib mesylate and how well it works in treating patients with myelofibrosis.

Condition or disease Intervention/treatment Phase
Chronic Myeloproliferative Disorders Drug: imatinib mesylate Phase 2

Detailed Description:



  • Determine the safety, efficacy, and tolerability of imatinib mesylate in patients with myelofibrosis with myeloid metaplasia.
  • Determine the 3-, 6-, and 12-month major and minor erythroid response rates in patients treated with this drug.


  • Determine reduction in marrow fibrosis in patients treated with this drug.
  • Determine decrease in spleen size in patients treated with this drug.

OUTLINE: This is a multicenter, open-label, nonrandomized, pilot study.

Patients receive oral imatinib mesylate once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients who do not experience a minor erythroid response or a 50% reduction in spleen size after 6 months of treatment are removed from the study. Patients experiencing clinical benefit (e.g., ongoing erythroid response) after 1 year of treatment may continue treatment with imatinib mesylate as above at the discretion of the principal investigator.

PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Pilot Study to Determine the Safety and Preliminary Efficacy of Imatinib Mesylate (Gleevec) in Patients With Myelofibrosis With Myeloid Metaplasia
Study Start Date : August 2005
Actual Primary Completion Date : March 2007
Actual Study Completion Date : October 2011

Intervention Details:
  • Drug: imatinib mesylate
    Once daily oral administration of Imatinib Mesylate at a dose of 600mg for 12 months.

Primary Outcome Measures :
  1. Percentage of Participants With Major and/or Minor Erythroid Responses at 3, 6, and 12 Months of Therapy [ Time Frame: At 3,6, and 12 months of therapy ]

    A major response = transfusion independent or a>2.0g/dl rise in hemoglobin without transfusion maintained for at least 8 weeks.

    Minor response= > 1 to 2.0g/dl incremental rise in hemoglobin maintained for at lease 8 weeks with a decrease in transfusion requirements of at least 50% compared to the mean transfusion requirement during the 8 week pre-study period.

Secondary Outcome Measures :
  1. Reduction in Marrow Fibrosis and Decrease in Spleen Size [ Time Frame: After 6 and 12 months of therapy ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of myelofibrosis with myeloid metaplasia (MMM), defined by all of the following:

    • Leukoerythroblastic blood picture
    • Fibrosis involving > 1/3 sectional area of bone marrow biopsy
    • Splenomegaly (unless patient has undergone prior splenectomy)
    • Philadelphia chromosome negative
    • No myelodysplastic syndrome
    • No systemic disorders associated with marrow fibrosis
  • Red blood cell transfusion dependent, defined by 1 of the following:

    • Patient has required ≥ 2 units of red blood cells every 4 weeks within the past 8 weeks
    • Hemoglobin ≤ 8 g/dL on ≥ 3 occasions (≥ 2 weeks apart ) over the past 8 weeks
  • No evidence of disease transformation to acute myelogenous leukemia, defined as > 20% blasts in bone marrow and/or peripheral blood


Performance status

  • ECOG 0-3

Life expectancy

  • Not specified


  • Absolute neutrophil count > 1,000/mm^3
  • Platelet count > 50,000/mm^3


  • Bilirubin ≤ 1.5 times upper limit of normal (ULN)
  • AST or ALT ≤ 2 times ULN (unless due to extramedullary hematopoiesis in the liver)


  • Creatinine ≤ 1.5 times ULN


  • No New York Heart Association grade III-IV heart disease


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective barrier method contraception during and for 3 months after completion of study treatment
  • No serious, uncontrolled medical condition
  • No patients who are considered potentially unreliable or with a history of noncompliance to medical regimens


Biologic therapy

  • More than 2 weeks since prior interferon alfa


  • No concurrent chemotherapy except hydroxyurea to control elevated blood counts

Endocrine therapy

  • More than 4 weeks since prior corticosteroids, danazol, or other androgens for MMM


  • More than 4 weeks since other prior treatment for MMM
  • No other concurrent experimental drug therapy for MMM

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 identifier (NCT number): NCT00245128

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United States, Oregon
OHSU Knight Cancer Institute
Portland, Oregon, United States, 97239-3098
Sponsors and Collaborators
OHSU Knight Cancer Institute
National Cancer Institute (NCI)
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Principal Investigator: Michael Mauro, MD OHSU Knight Cancer Institute

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Responsible Party: OHSU Knight Cancer Institute Identifier: NCT00245128     History of Changes
Other Study ID Numbers: CDR0000445435
OHSU-541 ( Other Identifier: OHSU IRB )
OHSU-HEM-01071-L ( Other Identifier: OHSU Knight Cancer Institute )
First Posted: October 27, 2005    Key Record Dates
Results First Posted: January 13, 2012
Last Update Posted: January 13, 2012
Last Verified: December 2011
Keywords provided by OHSU Knight Cancer Institute:
chronic idiopathic myelofibrosis
polycythemia vera
essential thrombocythemia
Additional relevant MeSH terms:
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Primary Myelofibrosis
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Imatinib Mesylate
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action