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A Phase 2 Study of Azacitidine in Chronic Myelomonocytic Leukemia (CMML)

Phase 2
18 Years
Open (Enrolling)

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Trial Information

A Phase 2 Study of Azacitidine in Chronic Myelomonocytic Leukemia (CMML)



- 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

Inclusion Criteria


- 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

- Red cell transfusion dependence and pre-transfusion hemoglobin < 9.0

- 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

- 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

Type of Study:


Study Design:

Allocation: Non-Randomized, Primary Purpose: Treatment

Outcome Measure:

Safety and tolerability

Safety Issue:


Principal Investigator

David T. Bowen, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Leeds Cancer Centre at St. James's University Hospital



Study ID:




Start Date:

January 2010

Completion Date:

Related Keywords:

  • Leukemia
  • chronic myelomonocytic leukemia
  • Leukemia
  • Leukemia, Myelomonocytic, Chronic
  • Leukemia, Myelomonocytic, Acute