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Azacitidine in Treating Patients With Chronic Myelomonocytic Leukemia

This study has been completed.
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: November 4, 2010
Last updated: August 23, 2013
Last verified: November 2010

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

PURPOSE: This phase II trial is studying the side effects of azacitidine and to see how well it works in treating patients with chronic myelomonocytic leukemia.

Condition Intervention Phase
Drug: azacitidine
Other: laboratory biomarker analysis
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Primary Purpose: Treatment
Official Title: A Phase 2 Study of Azacitidine in Chronic Myelomonocytic Leukemia (CMML)

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Safety and tolerability
  • Overall response rate

Secondary Outcome Measures:
  • Incidence of clinical remission/complete remission or partial response according to International Working Group (IWG) criteria
  • Hematological improvement according to IWG criteria
  • Overall survival
  • Progression-free survival
  • Time to acute myeloid leukemia (AML) transformation of CMML
  • Time to death or AML transformation of CMML
  • Biological correlates

Estimated Enrollment: 30
Study Start Date: January 2010
Study Completion Date: May 2013
Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Detailed Description:



  • To assess the safety and tolerability of azacitidine in patients with chronic myelomonocytic leukemia (CMML).
  • To assess the overall response rate in these patients.


  • To assess the incidence of clinical remission/complete remission or partial response in these patients.
  • To assess hematological improvement in patients treated with this drug.
  • To assess the overall survival of patients treated with this drug.
  • To assess progression-free survival of patients treated with this drug.
  • To assess the time to acute myeloid leukemia (AML) transformation of CMML.
  • To assess the time to death or AML transformation of CMML.
  • To assess the biological correlates.

OUTLINE: This is a multicenter study.

Patients receive azacitidine subcutaneously on days 1-5 and 8-9. Treatment repeats every 4 weeks for at least 6 courses in the absence of loss of response/disease progression or unacceptable toxicity. Patients undergo response evaluation after 6 courses or the last course of treatment. Responders may continue azacitidine until loss of response/disease progression or unacceptable toxicity.

Some patients undergo blood, bone marrow, and buccal swab sample collection periodically for correlative studies.

After completion of study treatment, patients are followed up for 1 month.

Peer Reviewed and Funded or Endorsed by Cancer Research UK


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


  • Diagnosis of 1 of the following:

    • All chronic myelomonocytic leukemia (CMML)-2 patients
    • CMML-1 patients meeting any of the following criteria:

      • Symptomatic bone marrow failure/myeloproliferation defined as any of the following:

        • Red cell transfusion dependence and pre-transfusion hemoglobin < 9.0 g/dL
        • Symptomatic anemia (hemoglobin < 11.5 g/dL)
        • Thrombocytopenia (platelet count < 50 x 10^9/L)
        • Symptomatic bleeding due to platelet functional defect or disseminated intravascular coagulation (DIC)/fibrinolysis
        • White cell count (WCC) > 50 x 10^9/L
      • Düsseldorf Score of intermediate or high risk for proliferative CMML-1 (i.e., WCC > 12 x 10^9/L)
      • International Prognostic Scoring System (IPSS) score of intermediate-2 or high risk for non-proliferative CMML-1 (i.e., WCC < 12 x 10^9/L)
      • Systemic symptoms including weight loss with no alternative explanation (10% of baseline weight within the past 6 months)
      • Symptomatic splenomegaly
      • Symptomatic extramedullary involvement (e.g. skin infiltration or serous effusions)
  • No CMML with eosinophilia and 5q33 abnormality


  • WHO performance status 0-2
  • Creatinine ≤ 2 times upper limit of normal
  • Not pregnant or nursing
  • Negative urine pregnancy test
  • Fertile patients must use at least 2 forms of effective contraception during study and for 3 months after completion of study therapy
  • No other active malignant disease including basal cell or squamous cell carcinoma of the skin
  • No known HIV or infectious hepatitis B or hepatitis C
  • No active infection
  • No known hypersensitivity to azacitidine or mannitol


  • At least 28 days since other prior experimental drug or therapy
  • No prior chemotherapy for this disease except hydroxycarbamide
  • No other concurrent anticancer or investigational agents
  Contacts and Locations
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Please refer to this study by its identifier: NCT01235117

United Kingdom
Leeds Cancer Centre at St. James's University Hospital
Leeds, England, United Kingdom, LS9 7TF
Beatson West of Scotland Cancer Centre
Glasgow, Scotland, United Kingdom, G12 0YN
Sponsors and Collaborators
University of Leeds
Principal Investigator: David T. Bowen, MD Leeds Cancer Centre at St. James's University Hospital
  More Information Identifier: NCT01235117     History of Changes
Other Study ID Numbers: CDR0000688119
Study First Received: November 4, 2010
Last Updated: August 23, 2013

Keywords provided by National Cancer Institute (NCI):
chronic myelomonocytic leukemia

Additional relevant MeSH terms:
Leukemia, Myelomonocytic, Acute
Leukemia, Myelomonocytic, Chronic
Neoplasms by Histologic Type
Leukemia, Myeloid
Myelodysplastic-Myeloproliferative Diseases
Bone Marrow Diseases
Hematologic Diseases
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors processed this record on May 25, 2017