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Everolimus in Combination With Exemestane in the Treatment of Postmenopausal Women With Estrogen Receptor Positive Locally Advanced or Metastatic Breast Cancer Who Are Refractory to Letrozole or Anastrozole

Phase 3
Completed
Conditions
Breast Cancer
Interventions
Registration Number
NCT00863655
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

There are no treatments specifically approved after recurrence or progression on a non steroidal aromatase inhibitors (NSAI). In light of the need for new treatment options for postmenopausal women after failure of prior NSAI therapy, the purpose of this Phase III study is to compare efficacy and safety of a treatment with exemestane + everolimus to exemestane + placebo in postmenopausal women with estrogen receptor positive locally advanced or metastatic breast cancer refractory to NSAI.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
724
Inclusion Criteria
  • Adult women (≥ 18 years of age) with metastatic or locally advanced breast cancer not amenable to curative treatment by surgery or radiotherapy.
  • Histological or cytological confirmation of estrogen-receptor positive (ER+) breast cancer
  • Postmenopausal women.
  • Disease refractory to non steroidal aromatase inhibitors (NSAI),
  • Radiological or clinical evidence of recurrence or progression on or after the last systemic therapy prior to randomization.
  • Patients must have at least one lesion that can be accurately measured or bone lesions in the absence of measurable disease as defined above.
Exclusion Criteria
  • HER2-overexpressing patients
  • Patients with only non-measurable lesions other than bone metastasis (e.g. pleural effusion, ascites etc.).
  • Patients who received more than one chemotherapy line for Advanced Breast Cancer.
  • Previous treatment with exemestane or mTOR inhibitors.
  • Known hypersensitivity to mTOR inhibitors, e.g. sirolimus (rapamycin).
  • Radiotherapy within four weeks prior to randomization
  • Currently receiving hormone replacement therapy,

Other protocol-defined inclusion/exclusion criteria may apply

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Placebo + ExemestaneEverolimus PlaceboPlacebo of everolimus in combination with exemestane 25 mg daily
Everolimus + ExemestaneEverolimusEverolimus 10 mg daily in combination with exemestane 25 mg daily
Everolimus + ExemestaneExemestaneEverolimus 10 mg daily in combination with exemestane 25 mg daily
Placebo + ExemestaneExemestanePlacebo of everolimus in combination with exemestane 25 mg daily
Primary Outcome Measures
NameTimeMethod
Progression-free Survival (PFS) Based on Local Radiology Review of Tumor Assessments.date of randomization to the date of first documented tumor progression or death from any cause, whichever occurs first, reported between day of first patient randomized up to about 19 months

Progression-free survival, the primary endpoint in this study, is defined as the time from the date of randomization to the date of first documented radiological progression or death due to any cause. Disease progression was based on the tumor assessment by the local radiologist or investigator using RECIST 1.0 criteria. If a patient did not progress or known to have died at the date of the analysis cut-off or start of another antineoplastic therapy, the PFS date was censored to the date of last adequate tumor assessment prior to cut-off date or start of antineoplastic therapy. For patients with lytic or mixed (lytic+sclerotic) bone lesions, the following is considered progression: appearance of ≥1 new lytic lesions in bone; the appearance of ≥ new lesions outside of bone and unequivocal progression of existing bone lesions.

Secondary Outcome Measures
NameTimeMethod
Overall Survival (OS) by Number of Deathsup to 53 months

Overall survival, the key secondary endpoint in this study, is defined as the time from date of randomization to the date of death due to any cause. If a patient is not known to have died, survival was censored at the date of last contact.

Overall Survival (OS) by Medianup to 53 months

Overall survival, the key secondary endpoint in this study, is defined as the time from date of randomization to the date of death due to any cause.

Overall Response Rate (ORR)up to 21 months

Overall response rate (ORR) is the percentage of patients with a best overall response of complete response (CR) or partial response (PR) according to RECIST 1.0. Per RECIST criteria 1.0, CR: Disappearance of all target lesions; PR: At least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the baseline sum of the longest diameters.

Clinical Benefit Rate (CBR)up to 21 months

CBR is defined as the percentage of patients with best overall response of either complete response (CR), a partial response (PR) or stable disease (SD) \>= 24 weeks, according to RECIST 1.0. Per RECIST criteria 1.0, CR: Disappearance of all target lesions; PR: At least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the baseline sum of the longest diameters; SD = Neither sufficient shrinkage to qualify for PR or CR nor an increase in lesions which would qualify for PD; PD = At least a 20% increase in the sum of the longest diameter of all measured target lesions, taking as reference the smallest sum of longest diameter of all target lesions recorded at or after baseline.

Proportion of Patients With no Deterioration of Eastern Cooperative Oncology Group Performance Status (ECOG PS) Using Kaplan-Meier2, 4, 6, 9 months

The ECOG PS (Eastern Cooperative Oncology Group Performance Scale) is a standard criteria for measuring how treatment of cancer impacts level of functioning in terms of the ability to care for oneself, daily activity, \& physical ability (walking, working, etc.). Scale score ranges:0 to 5, 5 being the worst. Scale index: 0: Fully active, able to carry on all pre-disease performance without restriction. 1: Restricted in physically strenuous activity but ambulatory \& able to carry out work of a light or sedentary nature. 2: Ambulatory \& capable of all self-care but unable to carry out any work activities. Up \& about more than 50% of waking hours. 3: Capable of only limited self-care, confined to bed or chair more than 50% of waking hours. 4 - Completely disabled. Cannot carry on any self-care. Totally confined to bed or chair. 5 - Dead. A deterioration of ECOG is an increase of 1 of the ECOG PS without improvement back to initial level at a subsequent time of measurement.

Patient-reported Outcomes (PROs): Time to Deterioration of PRO Scores Using Kaplan Meier - EORTC QLQ-C30Up to 21 months

The QLQ-C30 is composed of both multi-item scales and single-item measures. These include 5 functional scales, 3 symptom scales, a global health status - QoL scale, and 6 single items. Each of the multi-item scales includes a different set of items - no item occurs in more than 1 scale. All of the scales measures range in score from 0 to 100. A high scale score = higher response level. Thus a high score for a functional scale represents a healthy level of function, a high score for the global health status / QoL represents a high quality of life but a high score for a symptom scale / item represents a high level of symptomatology / problems. The principle for scoring these scales: 1.) Estimate the average of the items that contribute to the scale = raw score. 2.) Linear transformation to standardize the raw score, so that scores range from 0 to 100; a higher score represents a higher ("better") level of functioning, or a higher ("worse") level of symptoms.

Proportion of Patients With Having no Overall Response Based on Investigator Assessment2, 4, 6, 9 months

overall response = complete response (CR) + partial response (PR) per RECIST 1.0 Time to overall response (CR or PR) based on investigator is the time between date of randomization/start of treatment until first documented response (CR or PR). This analysis included all patients/responders. Patients who did not achieve a confirmed PR or CR were censored at last adequate tumor assessment date when they did not progress. Per RECIST criteria 1.0, CR: Disappearance of all target lesions; PR: At least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the baseline sum of the longest diameters.

Duration of Response (Among Participants With Best Overall Response of CR or PR) Estimated Per Kaplan-Meier21 months

Duration of response of CR or PR based on investigator applies only to patients whose best overall response was CR or PR (RECIST 1.0). The start date was the date of first documented response (CR or PR) and the end date and censoring is defined the same as that for time to progression. Per RECIST criteria 1.0, CR: Disappearance of all target lesions; PR: At least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the baseline sum of the longest diameters.

Everolimus Concentrations at Week 4pre-dose, 2 hours post-dose

Characterize the pharmacokinetics (PK) of everolimus in combination with exemestane using Cmin (pre-dose) and C2h (post-dose) at week 4 in a small group of patients.

Exemestane Concentrations at Week 4predose, 2 hours post-dose

Characterize the PK of exemestane in combination with or without everolimus using Cmin and C2h at week 4 in a small group of patients.

Estradiol Plasma ConcentrationsBaseline, Week 4

Compare estradiol concentrations from baseline to week 4 in both treatment arms.

Trial Locations

Locations (64)

University of Texas Southwestern Medical Center SimmonsComprehensiveCancerCtr.

🇺🇸

Dallas, Texas, United States

Mercy Medical Center Medical Oncology & Hematology

🇺🇸

Baltimore, Maryland, United States

Crescent City Research Consortium, LLC Dept of Hem&Onc Specialist - 2

🇺🇸

Metairie, Louisiana, United States

Regional Cancer Care Associates Dept. of the CCHD

🇺🇸

Cherry Hill, New Jersey, United States

Hematology Oncology Clinic Hematology Oncology Clinic (2)

🇺🇸

Baton Rouge, Louisiana, United States

Maryland Hematology/Oncology Associates, P.A.

🇺🇸

Baltimore, Maryland, United States

Holy Cross Hospital Holy Cross

🇺🇸

Silver Spring, Maryland, United States

Kaiser Permanente Medical Group Kaiser Permanente-Moanalua M.C

🇺🇸

Anaheim, California, United States

Ironwood Cancer and Research Centers

🇺🇸

Chandler, Arizona, United States

Highlands Oncology Group DeptofHighlandsOncologyGrp(2)

🇺🇸

Fayetteville, Arkansas, United States

Comprehensive Blood and Cancer Center Dept. of CBCC (3)

🇺🇸

Bakersfield, California, United States

Cancer Care Associates Dept.ofCancerCareAssoc. (2)

🇺🇸

Fresno, California, United States

Grass Valley Hematology Oncology Medical Group Dept. of Grass Valley Hem/Onc

🇺🇸

Grass Valley, California, United States

Sharp Memorial Hospital SharpClinicalOncologyResearch

🇺🇸

San Diego, California, United States

Scripps Clinic SC

🇺🇸

La Jolla, California, United States

USC/Kenneth Norris Comprehensive Cancer Center Regulatory Contact 3

🇺🇸

Los Angeles, California, United States

The Angeles Clinic and Research Institute

🇺🇸

Los Angeles, California, United States

St Joseph Heritage Healthcare Dept. of RRMG (4)

🇺🇸

Santa Rosa, California, United States

Premiere Oncology/Pinnacle Oncology Hematology Dept.ofPremiereOncologyAZ

🇺🇸

Santa Monica, California, United States

Florida Cancer Research Institute

🇺🇸

Davie, Florida, United States

Florida Cancer Specialists DeptofFloridaCancerSpecialists

🇺🇸

Fort Myers, Florida, United States

Comprehensive Cancer Center - Boca Raton Deerfield Beach

🇺🇸

Boca Raton, Florida, United States

Memorial Hospital Memorial Cancer Institute

🇺🇸

Hollywood, Florida, United States

Palm Beach Cancer Institute

🇺🇸

West Palm Beach, Florida, United States

Georgia Cancer Specialists. Drug Ship

🇺🇸

Decatur, Georgia, United States

Florida Medical Clinic PA Dept.ofFloridaMedicalClinic

🇺🇸

Zephyrhills, Florida, United States

Hematology Oncology of Indiana

🇺🇸

Indianapolis, Indiana, United States

Rush University Medical Center Study Coordinator

🇺🇸

Chicago, Illinois, United States

Oncology Specialists, SC Dept.of Oncology Specialists

🇺🇸

Park Ridge, Illinois, United States

Horizon Oncology Center

🇺🇸

Lafayette, Indiana, United States

Weinberg Cancer Institute at Franklin Square Hospital

🇺🇸

Baltimore, Maryland, United States

Anne Arundel Health System Research Institute Wayson Pavilion

🇺🇸

Annapolis, Maryland, United States

Frederick Memorial Hospital Dept. of FMH-IRB

🇺🇸

Frederick, Maryland, United States

St. Louis Cancer & Breast Institute Dept.ofSt.LouisCancer&Breast

🇺🇸

St. Louis, Missouri, United States

Lahey Clinic Dept of Lahey Clinic (2)

🇺🇸

Burlington, Massachusetts, United States

Fairview Southdale Medical Oncology Clinic

🇺🇸

Edina, Minnesota, United States

Southeast Nebraska Oncology Cancer Center

🇺🇸

Lincoln, Nebraska, United States

Trinitas Comprehensive Cancer Center Dept. of Trinitas

🇺🇸

Elizabeth, New Jersey, United States

University of New Mexico Cancer Research Center Dept of UNM Cancer & Research

🇺🇸

Albuquerque, New Mexico, United States

Clinical Research Alliance Dept.ofArenaOncologyAssoc(2)

🇺🇸

Lake Success, New York, United States

ProHealth Care

🇺🇸

Lake Success, New York, United States

Beth Israel Medical Center Dept.ofBeth Israel Med. Ctr(2)

🇺🇸

New York, New York, United States

Cancer Center of Kansas Dept.ofCancerCtr.ofKansas

🇺🇸

Wichita, Kansas, United States

University of California San Francisco UCSF Medical Center

🇺🇸

San Francisco, California, United States

Cancer Care Associates SC

🇺🇸

Tulsa, Oklahoma, United States

Marion L. Shepard Cancer Center

🇺🇸

Washington, North Carolina, United States

Penn State University / Milton S. Hershey Medical Center Division of Oncology (2)

🇺🇸

Hershey, Pennsylvania, United States

Weill Cornell Medical College Weill Cornell Med. Ctr.

🇺🇸

New York, New York, United States

Sarah Cannon Research Institute Dept.ofSarahCannonCancerCtr(5)

🇺🇸

Nashville, Tennessee, United States

Hematology Oncology Association of Rockland

🇺🇸

Nyack, New York, United States

Cancer Centers of Southwest Oklahoma Cancer Research Dept.of Southwest Oklahoma

🇺🇸

Lawton, Oklahoma, United States

The Center for Cancer and Blood Disorders Dept. of The Ctr for C & BD(2)

🇺🇸

Fort Worth, Texas, United States

University of Texas/MD Anderson Cancer Center Dept.ofMDAndersonCancerCtr(2)

🇺🇸

Houston, Texas, United States

Novartis Investigative Site

🇬🇧

Southampton, United Kingdom

Northern Utah Cancer Associates SC

🇺🇸

Ogden, Utah, United States

Utah Cancer Specialists Dept.of Utah Cancer Spec. (2)

🇺🇸

Salt Lake City, Utah, United States

Hope Oncology HOPE Richardson

🇺🇸

Richardson, Texas, United States

University of Utah / Huntsman Cancer Institute Dept.ofHuntsmanCancerInst.(2)

🇺🇸

Salt Lake City, Utah, United States

Central Utah Clinic CRAD001Y2301

🇺🇸

Provo, Utah, United States

Medical Oncology & Hematology Associates of Northern VA Med. Onc&Hem Assoc. of No.VA

🇺🇸

Reston, Virginia, United States

University of Wisconsin Hospital & Clinics UW ComprehensiveCancerCtr(2)

🇺🇸

Madison, Wisconsin, United States

MD Anderson Cancer Center - Orlando Dept.ofMDACC-Orlando(2)

🇺🇸

Orlando, Florida, United States

Medical University of South Carolina -Hollings Cancer Center Dept. MUSC/HollingsCancerCtr

🇺🇸

Charleston, South Carolina, United States

University of Louisville / James Graham Brown Cancer Center SC

🇺🇸

Louisville, Kentucky, United States

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