Clinical Trial: Tipifarnib in Treating Older Patients With Acute Myeloid Leukemia

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

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

Brief Summary: 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.

Detailed Summary:

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.

Current Primary Outcome: Complete Remission (CR) Rate [ Time Frame: From first treatment through follow up period, an expected average of 12 months ]

Original Primary Outcome: Number of Participants with Complete Remission [ Time Frame: From first treatment through follow up period, an expected average of 12 months ]

Current Secondary Outcome:

• Median Overall Survival (OS) [ Time Frame: From first treatment through follow up period, an expected average of 12 months ]
  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: 1 year ]
  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.
• Number of Participants With Relapse Free Survival [ Time Frame: 7 months ]
  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.

Original Secondary Outcome:

• Median Overall Survival (OS) Rate [ Time Frame: From first treatment through follow up period, an expected average of 12 months ]
  Overall survival is calculated from the first day of R115777 treatment and lasts until the date of death recorded on the CRF.
• Median 1-Year Survival Rate [ Time Frame: Up to 12 months per participant ]
  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 ]
  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.

Dates:
Date Received: May 24, 2011
Date Started: May 2011
Date Completion:
Last Updated: March 19, 2015
Last Verified: February 2015