Clinical Trial: Tipifarnib in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

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

Official Title: Phase I Trial of R115777 (NSC 702818) in Relapsed, Refractory or High Risk Myeloid Leukemia

Brief Summary: Tipifarnib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. This phase I trial is studying the side effects and best dose of tipifarnib in treating patients with relapsed or refractory acute myeloid leukemia

Detailed Summary:

PRIMARY OBJECTIVES:

I. To define the maximum tolerated dose (MTD) of R115777 (tipifarnib) in patients with relapsed, refractory, or high risk myeloid leukemias treated according to this regimen.

II. To assess the toxicity and preliminary assessment of efficacy of R115777 in patients with relapsed, refractory, or high risk myeloid leukemias.

OUTLINE: This is a dose-escalation, multicenter study.

Patients receive oral tipifarnib twice daily on days 1-7 and 15-21. Courses repeat every 28 days in the absence of unacceptable toxicity or disease progression. Patients achieving a complete response (CR) receive 2 additional courses beyond CR. Patients experiencing relapse after previously achieving CR may receive additional tipifarnib at the current dose level for newly registered patients.

Cohorts of 3-6 patients receive escalating doses of tipifarnib until the MTD is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

Patients are followed every 6 months for survival.

PROJECTED ACCRUAL: A total of 20-30 patients will be accrued for this study.

Sponsor: National Cancer Institute (NCI)

Current Primary Outcome:

• MTD defined as the highest dose level in which six patients have been evaluated for toxicity with no more than one patient experiencing dose limiting toxicity (DLT) assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 [ Time Frame: 28 days ]
  The toxicities observed at each dose level will be summarized in terms of type, severity, time of onset, duration, and reversibility or outcome. Tables will be created to summarize these toxicities and side effects by dose and by course.
• Objective tumor response [ Time Frame: Up to 6.5 years ]
  Will be summarized at each dose level, and the number and percent responding combined across dose levels.
• Survival [ Time Frame: From registration to time of death due to any cause, assessed up to 6.5 years ]
  Will be summarized with Kaplan-Meier plots.
• Time to treatment failure [ Time Frame: From registration to the first observation of disease progression, death due to any cause, or early discontinuation of treatment, assessed up to 6.5 years ]
  Will be summarized with Kaplan-Meier plots.
• Duration of response [ Time Frame: Up to 6.5 years ]
  Will be summarized with Kaplan-Meier plots.

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: National Cancer Institute (NCI)

Dates:
Date Received: January 7, 2005
Date Started: October 2004
Date Completion:
Last Updated: February 1, 2013
Last Verified: January 2013