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Everolimus and Imatinib Mesylate in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia Who Are Not in Complete Cytogenetic Remission After Previous Imatinib Mesylate

Phase 1
Completed
Conditions
Leukemia
Registration Number
NCT00093639
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

RATIONALE: Drugs used in chemotherapy, such as everolimus, work in different ways to stop cancer cells from dividing so they stop growing or die. Imatinib mesylate may stop the growth of cancer cells by blocking the enzymes necessary for their growth. Combining everolimus with imatinib mesylate may be effective in killing cancer cells that have become resistant to imatinib mesylate.

PURPOSE: This phase I/II trial is studying the side effects and best dose of everolimus when given together with imatinib mesylate and to see how well they work in treating patients with chronic phase chronic myelogenous leukemia who are not in complete cytogenetic remission after previous imatinib mesylate.

Detailed Description

OBJECTIVES:

Primary

* Determine the safety, tolerability, and biological activity of everolimus when combined with imatinib mesylate in patients with chronic phase chronic myelogenous leukemia that is not in complete cytogenetic remission after prior imatinib mesylate. (Phase I)

* Determine, preliminarily, the clinical efficacy of this regimen, in terms of 3-month improvement by at least one cytogenetic category and the duration of cytogenetic improvements, in these patients. (Phase II)

Secondary

* Determine the 6-month rate of cytogenetic improvements in patients treated with this regimen.

* Determine the rate of confirmed cytogenetic improvements in patients treated with this regimen.

* Determine the rate and duration of major cytogenetic response in patients treated with this regimen.

* Determine the rate and kinetics of molecular response in patients treated with this regimen.

* Correlate genetic variation in drug metabolism genes, leukemia genes, and drug target genes with response in patients treated with this regimen.

* Determine the pharmacokinetics of this regimen in these patients.

* Determine whether the mTOR pathway activity, as determined by molecular pathologic examination before and during treatment with this regimen, is predictive of response in these patients.

OUTLINE: This is a phase I, non-randomized, open-label, multicenter, dose-escalation study of everolimus followed by a phase II study. Patients are stratified according to baseline cytogenetic status (Philadelphia chromosome-positive cells in bone marrow) (\>0% and ≤ 95% vs \> 95%).

* Phase I: Patients receive oral everolimus once daily (or once weekly) and oral imatinib mesylate once daily beginning on day 1. Treatment continues in the absence of disease progression or unacceptable toxicity.

Cohorts of 4-6 patients receive escalating doses of everolimus until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose-limiting toxicity.

* Phase II: Patients receive everolimus and imatinib mesylate as in phase I at the MTD.

Patients are followed every 6 months.

PROJECTED ACCRUAL: A total of 4-98 patients (4-34 for phase I and up to 64 for phase II \[34 patients with \> 0% and ≤ 95% Philadelphia chromosome (Ph)-positive cells and 30 patients with \> 95% Ph-positive cells\]) will be accrued for this study.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
Not specified
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Tolerability and biological activity of everolimus and imatinib mesylate every 6 months after completion of study treatment
Secondary Outcome Measures
NameTimeMethod
Cytogenetic improvements at 3 and 6 months and then every 6 months after completion of study treatment
Changes in the amounts of the Bcr-Abl transcripts as measured by quantitative real time reverse transcriptase PCR (QT-PCR) every 6 months after completion of study treatment
mTOR pathway activity at baseline and during treatment measured by molecular pathological examination of blood and bone marrow cells every 6 months after completion of study treatment
Disease-related mutations and gene expression changes in blood, bone marrow cells, and in plasma every 6 months after completion of study treatment
Effects of genetic variation in drug metabolism genes, leukemia genes, and drug target genes on patient response measured every 6 months after completion of study treatment
Pharmacokinetics of combination everolimus and imatinib every 6 months after completion of study treatment

Trial Locations

Locations (1)

Roswell Park Cancer Institute

🇺🇸

Buffalo, New York, United States

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