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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
37
60mg orally 2 times a day for 28 days
80mg 2 times a day, dependent upon response to cycle 1
40mg 2 times a day, dependent upon response to cycle 1
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.
Time frame: 56 days
Number of days to response
Response as defined by CR, CTC, or HR.
Time frame: 56 Days
Number of days to disease progression
Response as defined by CR, CTC, or HR.
Time frame: 56 Days
Levels of FLT-3 phosphorylation inhibition in vivo
The degree of inhibition of FLT-3 autophosphorylation measured in ex vivo bioassay plasma samples
Time frame: 56 Days
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