Phase I Study of the Farnesyl Transferase Inhibitor R115777 (NSC #702818) in Patients With Myelodysplastic Syndrome
I. To determine the toxicity profile and antitumor activity of the farnesyltransferase
(FTase) inhibitor R115777 (tipifarnib) in patients with myelodysplastic syndrome (MDS)
treated on a one week on/one week off schedule.
II. To determine the effect on R115777 on a one week on/one week off schedule on FTase
activity, prenylation of RAS and other substrates and on downstream effects.
OUTLINE: This is a dose-escalation study.
Patients receive tipifarnib orally (PO) twice daily (BID) on weeks 1, 3, 5, and 7. Treatment
repeats every 8 weeks for up to 2 courses in the absence of disease progression or
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
MTD defined as the next lower dose level at which 2 patients experience dose limiting toxicity (DLT) defined as grade 3 or 4 toxicity according to the Cancer Therapy Evaluation Program Common Toxicity Criteria
The final analysis will report all toxicities by grade, dose, cycle, and by cumulative dose.
Up to 8.5 years
M.D. Anderson Cancer Center
United States: Food and Drug Administration
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