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Azacitidine in Treating Patients With Chronic Myelomonocytic Leukemia

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

Status
Completed
Phases
Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT01235117
Enrollment
30
Registered
2010-11-05
Start date
2010-01-31
Completion date
2013-05-31
Last updated
2013-08-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Leukemia

Keywords

chronic myelomonocytic leukemia

Brief summary

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.

Detailed description

OBJECTIVES: Primary * To assess the safety and tolerability of azacitidine in patients with chronic myelomonocytic leukemia (CMML). * To assess the overall response rate in these patients. Secondary * 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

Interventions

DRUGazacitidine
OTHERlaboratory biomarker analysis

Sponsors

University of Leeds
Lead SponsorOTHER

Study design

Allocation
NON_RANDOMIZED
Primary purpose
TREATMENT

Eligibility

Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No

Inclusion criteria

DISEASE CHARACTERISTICS: * 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 PATIENT CHARACTERISTICS: * 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 PRIOR CONCURRENT THERAPY: * 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

Design outcomes

Primary

MeasureTime frame
Safety and tolerability
Overall response rate

Secondary

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

Countries

United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026