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

Phase 2

Completed

• Conditions: Leukemia, Myeloid
• Interventions: Drug: CEP-701 60mg; Drug: Cep-701 80mg; Drug: Cep-701 40mg

• Registration Number: NCT00030186
• Lead Sponsor: Cephalon

Brief Summary

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.

Detailed Description

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 Timeline

• Study Start: 2002-01
• Study First Submitted: 2002-02-07
• Study First Submitted QC: 2002-02-07
• Study First Posted: 2002-02-08
• Primary Completion: 2003-02
• Study Completion: 2003-02
• Disposition First Submitted: 2012-05-03
• Disposition First Submitted QC: 2012-05-03
• Disposition First Posted: 2012-05-08
• Status Verified: 2012-08
• Last Update Submitted: 2012-08-14
• Last Update Posted: 2012-08-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 37

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Cycle 1	CEP-701 60mg	60mg
Cycle 2	Cep-701 80mg	80mg dependent upon response to Cycle 1
Cycle 2b	Cep-701 40mg	40mg dependent upon response to Cycle 1

Primary Outcome Measures

Name	Time	Method
Blood Samples and/or bone marrow assessments for complete remission (CR), complete tumor clearance (CTC), or hematologic response (HR) from bone marrow	56 days	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.

Secondary Outcome Measures

Name	Time	Method
Number of days to response	56 Days	Response as defined by CR, CTC, or HR.
Number of days to disease progression	56 Days	Response as defined by CR, CTC, or HR.
Levels of FLT-3 phosphorylation inhibition in vivo	56 Days	The degree of inhibition of FLT-3 autophosphorylation measured in ex vivo bioassay plasma samples