Phase 2 Trial of R115777 in Previously Untreated Older Adults With AML and Baseline Presence of a Specific 2-Gene Expression Signature Ratio
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.
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.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Complete remission (complete and partial response, or stable disease) rate in patients with AML treated with tipifarnib
The exact 95% confidence interval of the response rate will be reported.
Up to 3 years
H. Lee Moffitt Cancer Center and Research Institute
United States: Food and Drug Administration
|Johns Hopkins University||Baltimore, Maryland 21205|
|Memorial Sloan Kettering Cancer Center||New York, New York 10021|
|H. Lee Moffitt Cancer Center and Research Institute||Tampa, Florida 33612|
|Weill Medical College of Cornell University||New York, New York 10021|
|Blood and Marrow Transplant Group of Georgia||Atlanta, Georgia 30342-1601|
|Emory University||Atlanta, Georgia 30322|