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A Phase 2 Study of Tipifarnib in Subjects with Chronic Myelomonocytic Leukemia
The main purpose of this study is to see how effective an investigational new drug (tipifarnib, the study drug) is to treat this cancer. Tipifarnib is a study drug that works inside some cancer cells leading them to die or to stop them from growing. "Investigational" means that tipifarnib has not yet been approved by regulatory authorities such as the United States Food and Drug Administration (FDA) for use outside of clinical trials. Other reasons for conducting the study are: - To determine for how long tipifarnib may reduce the size or slow down the growth of your tumor. - To determine the side effects of tipifarnib. - To study your cancer and determine why it responds or not to treatment with tipifarnib.
Primary Objective: To assess the antitumor activity of tipifarnib, in terms of Objective Response Rate (ORR), of tipifarnib in subjects with chronic myelomonocytic leukemia (CMML) and in subjects with CMML whose disease is KRAS/NRAS wild type. Secondary Objective: To assess the effect of tipifarnib on the following: Rate of complete response (CR), complete cytogenetic remission, partial remission, marrow response, and clinical benefit. Duration of Response. Rate of progression free survival (PFS) at 1 year. Rate of survival at 1 year. Adverse event (AE) profile according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.03 (NCI CTCAE v 4.03). Exploratory Objective: To explore potential biomarkers and their association with clinical benefit from tipifarnib including cancer gene mutations, killer cell immunoglobulin-like receptor (KIR) genotyping, immune cell subsets and other candidate biomarkers of tipifarnib in bone marrow, blood and serum samples.