An Open-Label Study of CEP-701 in Patients With Refractory, Relapsed, or Poor Risk Acute Myeloid Leukemia Expressing FLT-3 Activating Mutations
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.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Blood Samples and/or bone marrow assessments for complete remission (CR), complete tumor clearance (CTC), or hematologic response (HR) from bone marrow
Response rate of patients with refactory, relapsed, or poor risk acute myeloid leukemia (AML) expressing Fms-like tyrosine kinase 3 (FLT-3) activating mutations. CR is defined as presence of less than 5% myeloblasts in bone marrow with normalized peripheral blood cell counts. HR is defined as a 50% or more decrease in the absolute number of peripheral blast count or at least a 50% reduction in bone marrow blasts.
United States: Food and Drug Administration