Clinical Trial: A Study of 5-Azacitidine (Vidaza®) in Patients With Chronic Myelomonocytic Leukemia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase II Study of the Efficacy, Safety and Determinants of Response to 5-Azacitidine (Vidaza®) in Patients With Chronic Myelomonocytic Leukemia (CMML)

Brief Summary:

The primary objective of this study is:

Response to treatment will be evaluated according to the revised International Working Group (IWG) categories natural history, hematologic improvement and cytogenetic response1;2. The primary objective is:

To determine the rate of complete hematologic response and hematologic improvement (according to IWG 2006 criteria) in CMML patients treated with 5-azacitidine.


Detailed Summary:

In this study, eligible patients with a confirmed diagnosis of CMML will be treated with 5-azacitidine to determine the rates of complete hematologic response, hematologic improvement, complete and partial cytogenetic response, and overall and progression free survival.

To develop biomarkers associated with response and gain insights into the mechanisms that determine response, gene expression profiling, genome-wide SNP array analysis, microRNA analysis, and DNA methylation analysis will be performed prior to therapy and at defined time points during the study. Phosphoproteomics profiling may be included in the analysis.


Sponsor: University of Utah

Current Primary Outcome: Percentage of Patients With Complete Hematologic Response (According to IWG 2006 Criteria) in CMML Patients Treated With 5-azacitidine. [ Time Frame: 24 months ]

Complete Hematologic Response is defined as: bone marrow evaluation shows <= 5% myeloblasts with normal maturation of all cells lines; peripheral blood evaluation shows hemoglobin >= 11 g/dL, neutrophils >= 1000/mL, platelets >= 100,000/mL, 0% blasts


Original Primary Outcome: To determine the rate of complete hematologic response and hematologic improvement (according to IWG 2006 criteria) in CMML patients treated with 5-azacitidine. [ Time Frame: 24 months ]

Current Secondary Outcome:

Original Secondary Outcome:

  • • To determine the rate of complete and partial cytogenetic response to 5-azacitidine in CMML patients with clonal chromosomal abnormalities. [ Time Frame: 24 months ]
  • • To determine the rate of overall and progression free survival at 24 months in CMML subjects treated with 5-azacitidine [ Time Frame: 24 months ]
  • • To determine the frequency of known leukemia-associated mutations in the population under study. [ Time Frame: 48 months ]
  • • To screen for genetic losses and gains in CMML cells compared to non-leukemic control cells from the same patient, using high density SNP array analysis. [ Time Frame: 48 months ]
  • • To determine the gene expression profile and promoter methylation profile of CMML cells prior to and on therapy with 5-azacitidine. [ Time Frame: 48 months ]
  • • To develop biomarkers of response to 5-azacitidine. [ Time Frame: 48 months ]


Information By: University of Utah

Dates:
Date Received: April 29, 2011
Date Started: April 2011
Date Completion:
Last Updated: May 10, 2016
Last Verified: May 2016