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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00030186

Enrollment

Registered

2002-02-08

Start date

2002-01-31

Completion date

2003-02-28

Last updated

2012-08-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Leukemia, Myeloid

Keywords

AML, FLT-3 mutation

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.

Interventions

DRUGCEP-701 60mg

60mg orally 2 times a day for 28 days

DRUGCep-701 80mg

80mg 2 times a day, dependent upon response to cycle 1

DRUGCep-701 40mg

40mg 2 times a day, dependent upon response to cycle 1

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026