This phase II trial is studying how well tipifarnib works in treating older patients with acute myeloid leukemia. Tipifarnib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
PRIMARY OBJECTIVES: I. To determine the complete remission (CR) rate in acute myeloid leukemia (AML) patients prospectively selected for tipifarnib (ZARNESTRA) treatment on the basis of a 2-gene signature (RASGRP1:APTX ratio) in bone marrow aspirates. SECONDARY OBJECTIVES: I. To determine the median overall and 1-year survival of patients treated with this regimen II. To determine the median relapse-free survival of patients treated with this regimen. III. To determine the safety of this regimen in these patients IV. To determine the immunophenotypic expression of RASGRP1 on baseline bone marrow blasts and assess correlation with PCR-based detection. OUTLINE: This is a multicenter study. Patients receive tipifarnib orally twice daily on days 1-21. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Bone marrow aspirate and/or biopsy are collected at baseline and on day 28 of course 1 and 2 for RasGRP1 protein expression analysis by qRT-PCR. After completion of study therapy, patients are followed up every 30 days.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
21
Given PO
Correlative studies
Moffitt Cancer Center
Tampa, Florida, United States
Emory University/Winship Cancer Institute
Atlanta, Georgia, United States
Blood and Marrow Transplant Group of Georgia
Atlanta, Georgia, United States
Johns Hopkins University/Sidney Kimmel Comprehensive Cancer Center
Baltimore, Maryland, United States
Memorial Sloan-Kettering Cancer Center
New York, New York, United States
Weill Medical College of Cornell University
New York, New York, United States
University of North Carolina
Chapel Hill, North Carolina, United States
Complete Remission (CR) Rate
Complete Remission (CR) rate in Acute Myelogenous Leukemia (AML) patients prospectively selected for R115777R115777 (ZARNESTRA) treatment on the basis of a 2-gene signature (RASGRP1:APTX ratio) in bone marrow aspirates. AML Complete Remission: Bone marrow aspiration - Less than 5% leukemic blasts, Auer rods not detected; Peripheral blood counts - Absolute neutrophil count \>/= 1,000/mm\^3, Platelet count \>/= 100,000/mm\^3, Leukemic blasts not present; Blood-product transfusion independence; Absence of extramedullary leukemia.
Time frame: From first treatment through follow up period, an expected average of 12 months
Median Overall Survival (OS)
Overall survival is calculated from the first day of R115777 treatment and lasts until the date of death recorded on the case report form (CRF).
Time frame: From first treatment through follow up period, an expected average of 12 months
Median 1-Year Survival Rate
Prior to the early discontinuation of the study (for not meeting the primary endpoint of at least 3 CR/CRi after 2 cycles), investigators had planned to calculate one year survival from Kaplan Meier estimates.
Time frame: 1 year
Number of Participants With Relapse Free Survival
Relapse-free survival is calculated from the date of documentation of complete remission/morphologic complete remission with incomplete blood count recovery (CR/CRi) until disease relapse or death from any cause.
Time frame: 7 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.