Zarnestra in Newly Diagnosed Acute Myelogenous Leukemia (AML)With 2 Gene Expression Signature Ratio
The purpose of this study is to determine the ability of a new test to predict whether patients will benefit from treatment with R115777 (ZARNESTRA, also called Tipifarnib). R115777 is an orally dosed, medication that inhibits the activity of the Farnesyl Transferase protein. Scientific experiments have suggested that R115777 can inhibit the function of proteins that play a role in the development of leukemia, and clinical studies of R115777 in humans has demonstrated that it can induce complete remissions (CR) in approximately 8-20% of patients with AML. R115777 is an investigational or experimental anticancer agent that has not yet been approved by the Food and Drug Administration for use in Acute Myelogenous Leukemia (AML).
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase 2 Trial of R115777 in Previously Untreated Older Adults With AML and Baseline Presence of a Specific 2-Gene Expression Signature Ratio|
- Number of Participants with Complete Remission [ Time Frame: From first treatment through follow up period, an expected average of 12 months ] [ Designated as safety issue: No ]To determine the complete remission (CR) rate in AML patients prospectively selected for R115777R115777 (ZARNESTRA) treatment on the basis of a 2-gene signature (RASGRP1:APTX ratio) in bone marrow aspirates. Patients will be periodically followed after their completion of the study to collect information on subsequent therapies and survival data.
- Median Overall Survival (OS) Rate [ Time Frame: From first treatment through follow up period, an expected average of 12 months ] [ Designated as safety issue: No ]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).
- Median 1-Year Survival Rate [ Time Frame: Up to 12 months per participant ] [ Designated as safety issue: No ]One year survival will be calculated from Kaplan Meier estimates.
- Number of Serious Adverse Events Reported That May be Related to Study Treatment [ Time Frame: From first treatment through follow up period, an expected average of 7 months ] [ Designated as safety issue: Yes ]All patients receiving R115777 will be evaluable for toxicity from the time of their first treatment with R115777, up through the 30-day follow up period after treatment termination, or until the initiation of subsequent therapy, whichever comes first.
|Study Start Date:||May 2011|
|Estimated Study Completion Date:||June 2014|
|Estimated Primary Completion Date:||June 2014 (Final data collection date for primary outcome measure)|
Experimental: R115777 Therapy
Each participant will begin R115777 treatment with an orally dosed regimen of 300 mg twice a day (BID) for the first 21 consecutive days of a 28-day cycle.
All screened participants with an RASGRP1:APTX ratio ≥ 5 will be eligible for a R115777 treatment course of a maximum of 6 cycles. Treatment will be administered on an outpatient basis.
This study will test the ability of a newly developed screening test, the RASGRP1:aprataxin (APTX) ratio, to identify patients who are more likely to benefit from R115777 therapy. RASGRP1 and APTX are genes that are expressed in leukemia cells. This test is performed on a sample of bone marrow tissue, and determines the ratio of RASGRP1 and APTX expression in the bone marrow. Ratios that are C5 are will be considered as a positive result, and these patients will be eligible for treatment with R115777. The ratio of C5 is expected to be found in about 30% of patients. This screening test is still considered an experimental procedure.
|Contact: Jeffrey Lancet, M.D.||email@example.com|
|Contact: Elyce Turbafirstname.lastname@example.org|
|United States, Florida|
|H. Lee Moffitt Cancer Center and Research Institute||Active, not recruiting|
|Tampa, Florida, United States, 33612|
|United States, Georgia|
|Emory - Winship Cancer Institute||Recruiting|
|Atlanta, Georgia, United States, 30322|
|Contact: Elliott Winton, M.D. 404-778-5851 email@example.com|
|Principal Investigator: Marth Arellano, M.D.|
|United States, Illinois|
|University of Chicago Medical Center||Active, not recruiting|
|Chicago, Illinois, United States, 60637|
|United States, Maryland|
|Johns Hopkins Sidney Kimmel Comprehensive Cancer Center||Recruiting|
|Baltimore, Maryland, United States, 21231|
|Contact: Kevin Meads 410-614-0488 firstname.lastname@example.org|
|Principal Investigator: Judith Karp, M.D.|
|University of Maryland Medical Center||Recruiting|
|Baltimore, Maryland, United States, 21201|
|Contact: Dina Ioffe 410-528-7828 email@example.com|
|Principal Investigator: Maria Baer, M.D.|
|United States, New Jersey|
|Cancer Institute of New Jersey||Active, not recruiting|
|New Brunswick, New Jersey, United States, 08901|
|United States, New York|
|Cornell - Weill Medical College of Cornell University||Active, not recruiting|
|New York, New York, United States, 10065|
|United States, North Carolina|
|UNC-Lineberger Comprehensive Cancer Center||Active, not recruiting|
|Chapel Hill, North Carolina, United States, 27599|
|United States, Tennessee|
|Vanderbilt-Ingram Cancer Center||Recruiting|
|Nashville, Tennessee, United States, 37232|
|Contact: Stephen Strickland, M.D. 615-936-0381 stephen.strickland@Vanderbilt.Edu|
|Principal Investigator: Stephen Strickland, M.D.|
|Principal Investigator:||Jeffrey Lancet, M.D.||H. Lee Moffitt Cancer Center and Research Institute|