A Phase 2 Study Evaluating the Efficacy of the Farnesyl Transferase Inhibitor (FTI) R115777 in Patients With Refractory or Relapsed Acute Myeloid Leukemia (AML)
Acute myeloid leukemia (AML) accounts for approximately 25% of all cases of leukemia
diagnosed in the Western hemisphere. First-line chemotherapy regimens induce complete
remissions in 55-75% of patients. However, between 50% and 70% of patients who achieve
remission will relapse. Current therapy for patients refractory to initial therapy or who
relapse within 6 months of remission is unsatisfactory because complete remission rates are
low and remission duration is brief. New drugs with novel mechanisms of action may be more
beneficial than the currently available ones. Tipifarnib represents a new class of oncology
drugs which have a specific cellular target (inhibition of the farnesyl transferase
protein), one of the components of the Ras oncogene, as its specific mechanism of acton. It
is believed that inhibition of this protein will lead to a decrease of cellular
proliferation or cell death. This is an open-label, multicenter, non-comparative phase 2
study investigating the efficacy and safety of farnesyl transferase inhibition with
tipifarnib administered orally as a single agent, twice daily, for the first 21 days of
every 28 day cycle. Patients are enrolled by disease status into two cohorts; Cohort 1
includes patients with relapsed AML and Cohort 2, patients with refractory AML. All patients
will be treated for a sufficient length of time to determine response to study medication
(effectiveness) by evaluating the rate of complete remission (CR) or complete remission with
incomplete platelet recovery (CRp), duration of complete remission, time to disease
progression and progression-free survival, overall survival, and to characterize clinical
benefit and quality of life (QOL). The safety profile of tipifarnib will also be determined
in patients with refractory of relapsed AML.. The patients will receive six tablets (100 mg
each) of tipifarnib twice daily for 21 of 28 day cycles (7 day rest period between cycles).
Patients may receive tipifarnib until disease progression or unacceptable toxicity occurs.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
The objective is to determine the rate of complete remission or complete remission with incomplete platelet recovery induced by tipifarnib in patients with refractory or relapsed AML treated until disease progression or unacceptable toxicity occurs.
Johnson & Johnson Pharmaceutical Research & Development, L.L. C. Clinical Trial
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
United States: Food and Drug Administration