A Phase 2 Study of Tipifarnib in Large Granular Lymphocyte (LGL) Leukemia
I. Estimate the complete response rate, partial response rate, and overall response rate in
patients with natural killer (NK)- or T-cell-large granular lymphocyte (LGL) leukemia who
present with neutropenia or anemia treated with tipifarnib.
I. Determine the toxicity of tipifarnib in these patients. II. Determine the mechanism of
treatment responses in these patients through correlative laboratory studies.
OUTLINE: Patients are stratified by disease type (natural killer-large granular lymphocyte
[LGL] leukemia vs T-cell-LGL leukemia).
Patients receive oral tipifarnib twice daily on days 1-21. Treatment repeats every 28 days
for 4 courses in the absence of disease progression or unacceptable toxicity. Patients are
evaluated after completion of course 4. Patients achieving complete response receive 1
additional course of treatment. Patients achieving partial response receive 4 additional
courses of treatment in the absence of disease progression or unacceptable toxicity.
Patients undergo blood collection periodically during study for response mechanism studies
and other biomarker correlative studies, including mutations of K-ras and N-ras genes.
After completion of study treatment, patients are followed every 6 months for 5 years.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Response rates to tipifarib defined as the proportion of patients achieving a complete response (CCR) or partial response (PR)
Up to 5 years
Case Comprehensive Cancer Center
United States: Food and Drug Administration
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