Open Study of CEP-701 in Patients With Refractory Acute Myeloid Leukemia With FLT-3 Mutation

An Open-Label Study of CEP-701 in Patients With Refractory, Relapsed, or Poor Risk Acute Myeloid Leukemia Expressing FLT-3 Activating Mutations

The purpose of this study is to determine the response rate of patients with refractory, relapsed or poor risk AML expressing FLT-3 activating mutations, when administered CEP-701 at a dosage of 60 mg 2 times a day.

This is an open-label study of CEP-701 in patients with refractory, relapsed, or poor risk AML expressing FLT-3 activating mutations. Patients who meet eligibility criteria will be enrolled at a dosage of 60 mg orally 2 times a day for 28 days (1 cycle). Upon completion of cycle 1, the dosage may be increased to 80mg 2 times a day or decreased to 40 mg 2 times a day, dependent upon response to 60 mg dosage.

INCLUSION CRITERIA:

• patient must have confirmed diagnosis of refractory or relapsed AML that expresses a FLT-3 mutation
• patient must have life expectancy of more than 2 months
• patient must be fully recovered from reversible side effects of previous therapy for cancer

EXCLUSION CRITERIA:

• total bilirubin, ALT or AST greater than 2 times upper limit of normal
• patient <65 years of age with estimated creatinine clearance less than 60 mL/min; patient >65 years of age with serum creatinine > 1.5 times the upper limit of normal (ULN)
• received any investigational drug within past 4 weeks
• GI disturbance/malabsorption that may affect absorption of CEP-701
• HIV positive
• received NSAID within prior 14 days
• has active infection