MedPath

Efficacy and Safety Study of Enzalutamide in Combination With Exemestane in Patients With Advanced Breast Cancer

Phase 2
Completed
Conditions
Breast Cancer
Interventions
Drug: Placebo (for enzalutamide)
Registration Number
NCT02007512
Lead Sponsor
Pfizer
Brief Summary

The purpose of this study is to determine if enzalutamide given in combination with exemestane is safe and effective in patients with advanced breast cancer.

Detailed Description

This is a Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Efficacy and Safety of Enzalutamide in Combination With Exemestane in Patients With Advanced Breast Cancer That Is Estrogen or Progesterone Receptor Positive and HER2-Normal.

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
247
Inclusion Criteria
  • Willing and able to provide informed consent;
  • Postmenopausal;
  • Advanced histologically confirmed breast cancer that is ER+, PgR+, or both, and HER-2 normal;
  • Up to one prior hormone therapy and up to one prior chemotherapy in the advanced setting is allowed;
  • Availability of a representative, formalin-fixed, paraffin-embedded tumor specimen that enabled the diagnosis of breast cancer with viable tumor cells in a tissue block or unstained serial slides accompanied bay an associated pathology report;
  • Measurable disease. Patients with non-measurable bone or skin disease as their only manifestation of advanced breast cancer are also eligible;
  • Eastern Cooperative Oncology Group (ECOG) status of 0 or 1;
Exclusion Criteria
  • Any severe concurrent disease, infection, or comorbid condition that renders the patient inappropriate for enrollment in the opinion of the investigator;
  • Any condition or reason that interferes with the patient's ability to participate in the trial, that may cause undue risk, or complicates the interpretation of safety data, in the opinion of the investigator;
  • Current or previously treated brain metastasis or leptomeningeal disease;
  • Prior therapy (> 28 days) with exemestane in the metastatic setting (Patients receiving exemestane in the adjuvant setting and having disease recurrence more than 1 year after treatment discontinuation are eligible);
  • Requires treatment for tuberculosis or HIV infection;
  • Radiation therapy within 7 days before randomization;
  • History of another invasive cancer within 5 years before randomization;
  • History of seizure or any condition that may predispose to seizure;
  • Clinically significant cardiovascular disease;
  • Active gastrointestinal disorder;
  • Major surgery within 28 days prior to randomization;
  • Treatment with any oral anticancer or with any non-hormonal anticancer agent within 14 days before randomization;
  • Treatment with any approved or investigational agent that blocks androgen synthesis or targets the androgen receptor;
  • Treatments with any of the following medications within 14 days before randomization: Estrogens, Androgens, or Systemic radionuclides;
  • Hypersensitivity reaction to exemestane.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Placebo & exemestaneexemestanePlacebo and exemestane 25mg (overencapsulated to match 50mg dose during the blinded portion of the study and one 25mg tablet without placebo after unblinding) once daily after food.
Placebo & exemestanePlacebo (for enzalutamide)Placebo and exemestane 25mg (overencapsulated to match 50mg dose during the blinded portion of the study and one 25mg tablet without placebo after unblinding) once daily after food.
Enzalutamide & exemestaneexemestaneEnzalutamide 160 mg/day administered as four 40mg soft gelatin capsules by mouth once daily with or without food and exemestane 50mg (two 25mg tablets overencapsulated as a single capsule during the blinded portion of the study and two 25mg tablets after unblinding) once daily after food.
Enzalutamide & exemestaneEnzalutamideEnzalutamide 160 mg/day administered as four 40mg soft gelatin capsules by mouth once daily with or without food and exemestane 50mg (two 25mg tablets overencapsulated as a single capsule during the blinded portion of the study and two 25mg tablets after unblinding) once daily after food.
Primary Outcome Measures
NameTimeMethod
Progression Free Survival (PFS): Diagnostic Positive (DX+) Population By Interactive Web Recognition System (IWRS)From randomization until PD, last tumor assessment without PD before new antitumor treatment initiation or death due to any cause, whichever occurred first (up to 3 years)

PFS was defined as the time in months from randomization to the first documentation of PD or death on study due to any cause, whichever occurred first. PD according to RECIST 1.1, was defined as \>= 20% increase in the sum of diameters of the target lesions taking as a reference the smallest sum recorded since the start of treatment or unequivocal progression in non-target lesions or the appearance of 1 or more new lesions. The analysis of PFS was based on investigator assessment of disease progression. Participants who were not known to have had a PFS event at the analysis date were censored at last tumor assessment date prior to data cutoff or date of new treatment initiation, whichever occurred first.

Progression Free Survival (PFS): Intent-to-Treat (ITT) Population By Interactive Web Recognition System (IWRS)From randomization until PD, last tumor assessment without PD before new antitumor treatment initiation or death due to any cause, whichever occurred first (up to 3 years)

PFS was defined as the time in months from randomization to the first documentation of progression of disease (PD) or death on study due to any cause, whichever occurred first. PD according to response evaluation criteria in solid tumors version 1.1 (RECIST 1.1) was defined as greater than or equal to (\>=) 20 percent (%) increase in the sum of diameters of the target lesions taking as a reference the smallest sum recorded since the start of treatment or unequivocal progression in non-target lesions or the appearance of 1 or more new lesions. The analysis of PFS was based on investigator assessment of disease progression. Participants who were not known to have had a PFS event at the analysis date were censored at last tumor assessment date prior to data cutoff or date of new treatment initiation, whichever occurred first.

Secondary Outcome Measures
NameTimeMethod
Clinical Benefit Rate-24 (CBR-24)From randomization up to 3 years

CBR-24: Percentage of participants with a best response of complete response (CR), partial response (PR), or stable disease (SD) sustained for atleast 24 weeks, as determined by investigator using RECIST 1.1. CR: Disappearance of all (target and non-target) lesions and normalization of tumor marker level for non-target lesions. All lymph nodes (target and non-target) must be non-pathological in size (less than \[\<\] 10 millimeter \[mm\] short axis). PR: \>=30% decrease in sum of diameters of target lesions, using baseline sum diameters as reference. SD: Neither sufficient reduction to qualify as PR nor sufficient increase to qualify as PD, using the smallest sum diameters during study as reference. PD: \>=20% increase (an absolute increase of \>=5 mm) in sum of diameters of target lesions, using the smallest sum during the study as a reference (including baseline sum), or unequivocal progression of existing non-target lesions, or appearance of atleast 1 new target or non-target lesions.

Concentration Versus Time Summary of ExemestanePredose, 1 and 6 hour postdose on Day 29, 57, 113 and 169

Concentration versus time summary was calculated by setting concentration values below limit of quantitation to zero.

Best Objective Response RateFrom randomization until CR or PR, whichever occurred first (up to 3 years)

Best objective response rate: Percentage of participants with measurable disease and with a best response of CR or PR according to RECIST 1.1. CR: Disappearance of all (target and non-target) lesions and normalization of tumor marker level for non-target lesions. All lymph nodes (target and non-target) must be non-pathological in size (\<10 mm short axis). PR: Atleast 30% decrease in sum of diameters of target lesions, using baseline sum diameters as reference. Response evaluation was based on investigators' judgment.

Time to ProgressionFrom randomization until PD or last tumor assessment without PD before new antitumor treatment initiation, whichever occurred first (up to 3 years)

Time to progression was defined as the time from the date of randomization to PD defined by the investigator using RECIST 1.1. PD: \>=20% increase (an absolute increase of \>=5 mm) in sum of diameters of target lesions, using the smallest sum during the study as a reference (including baseline sum), or unequivocal progression of existing non-target lesions, or appearance of atleast 1 new target or non-target lesions. Participants who did not experience disease progression, time to progression was right censored at the date of the last tumor assessment prior to data cutoff or date of new antitumor treatment, whichever occurred first.

Duration of Objective ResponseFrom first documentation of CR or PR until PD, or last tumor assessment without PD before new antitumor treatment initiation or death due to any cause, whichever occurred first (up to 3 years)

Duration of objective response: Time from first documentation of CR or PR, to the first documentation of PD or death due to any cause, whichever occurred first as determined by investigator using RECIST 1.1. CR: Disappearance of all (target and non-target) lesions and normalization of tumor marker level for non-target lesions. All lymph nodes (target and non-target) must be non-pathological in size (\<10 mm short axis). PR: \>=30% decrease in sum of diameters of target lesions, using baseline sum diameters as reference. PD: \>=20% increase (an absolute increase of \>=5 mm) in sum of diameters of target lesions, using the smallest sum during the study as a reference (including baseline sum), or unequivocal progression of existing non-target lesions, or appearance of atleast 1 new target or non-target lesions. Participants with no PD or death (after initial CR or PR) at the analysis date were censored at last tumor assessment date prior to date of new antitumor treatment or data cutoff.

Progression Free Survival (PFS) at 6 MonthsMonth 6

PFS at 6 months was defined as the percentage of participants with no event of disease progression at Month 6 landmark, estimated by Kaplan-Meier methods. PFS was defined as the time in months from randomization to the first documentation of PD or death on study due to any cause, whichever occurred first. PD: \>=20% increase (an absolute increase of \>=5 mm) in sum of diameters of target lesions, using the smallest sum during the study as a reference (including baseline sum), or unequivocal progression of existing non-target lesions, or appearance of atleast 1 new target or non-target lesions. The analysis of PFS was based on investigator assessment of disease progression.

Concentration Versus Time Summary of EnzalutamidePredose on Day 29, 57 and 113

Concentration versus time summary was calculated by setting concentration values below limit of quantitation to zero.

Time to ResponseFrom randomization until first documentation of CR or PR, or last tumor assessment without PD or death prior to new antitumor treatment initiation, whichever occurred first (up to 3 years)

Time to response: Time from randomization to first documentation of CR or PR. CR: Disappearance of all (target and non-target) lesions and normalization of tumor marker level for non-target lesions. All lymph nodes (target and non-target) must be non-pathological in size (\<10 mm short axis). PR: \>=30% decrease in sum of diameters of target lesions, using baseline sum diameters as reference. PD: \>=20% increase (an absolute increase of \>=5 mm) in sum of diameters of target lesions, using the smallest sum during the study as a reference (including baseline sum), or unequivocal progression of existing non-target lesions, or appearance of atleast 1 new target or non-target lesions. Participants who were not known to have had a CR or PR were censored at last tumor assessment date prior to data cutoff or date of new treatment initiation, whichever occurred first.

Concentration Versus Time Summary of N-desmethyl EnzalutamidePredose on Day 29, 57 and 113

N-desmethyl enzalutamide was the active metabolite of enzalutamide. Concentration versus time summary was calculated by setting concentration values below limit of quantitation to zero.

Trial Locations

Locations (79)

Indiana University Health Hospital

🇺🇸

Indianapolis, Indiana, United States

Indiana University Health Melvin and Bren Simon Cancer Center

🇺🇸

Indianapolis, Indiana, United States

Investigational Drug Services

🇺🇸

Indianapolis, Indiana, United States

Sidney and Lois Eskenazi Hospital

🇺🇸

Indianapolis, Indiana, United States

Springmill Medical Clinic

🇺🇸

Indianapolis, Indiana, United States

Brigham and Women's Hospital

🇺🇸

Boston, Massachusetts, United States

Dana-Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Minnesota Oncology Hematology, P.A

🇺🇸

Minneapolis, Minnesota, United States

Abbott Northwestern Hospital

🇺🇸

Minneapolis, Minnesota, United States

Allina Health System DBA Virginia Piper Cancer Institute

🇺🇸

Minneapolis, Minnesota, United States

Dr.Michaela Tsai

🇺🇸

Minneapolis, Minnesota, United States

The West Clinic, P.C.

🇺🇸

Corinth, Mississippi, United States

The West Clinic, P.C. d/b/a West Cancer Center

🇺🇸

Memphis, Tennessee, United States

Barnes-Jewish Hospital

🇺🇸

Saint Louis, Missouri, United States

Washington University Infusion Center Pharmacy

🇺🇸

Saint Louis, Missouri, United States

Washington University School of Medicine

🇺🇸

Saint Louis, Missouri, United States

Siteman Cancer Center-South County

🇺🇸

Saint Louis, Missouri, United States

Siteman Cancer Center- West County

🇺🇸

Saint Louis, Missouri, United States

Siteman Cancer Center

🇺🇸

Saint Peters, Missouri, United States

Hematology Oncology Associates of Northern NJ

🇺🇸

Morristown, New Jersey, United States

Henry-Joyce Cancer Clinic

🇺🇸

Nashville, Tennessee, United States

Investigational Products Center (IPC)

🇺🇸

Fort Worth, Texas, United States

Investigational Products Center(IPC)

🇺🇸

Fort Worth, Texas, United States

Investigational Products center

🇺🇸

Fort Worth, Texas, United States

lnvestigational Products Center (IPC)

🇺🇸

Fort Worth, Texas, United States

Texas Oncology - Memorial City

🇺🇸

Houston, Texas, United States

Sunnybrook Research Institute

🇨🇦

Toronto, Ontario, Canada

Dipartimento di Oncologia Medica, Istituto Nazionale Tumori Regina Elena

🇮🇹

Roma, Italy

Hospital Universitario HM Monteprincipe

🇪🇸

Boadilla del Monte, Madrid, Spain

Grupo Hospitalario Quiron - Hospital Universitari Quiron Dexeus

🇪🇸

Barcelona, Spain

Hospital Universitari Vall d'Hebron

🇪🇸

Barcelona, Spain

Brighton and Sussex University Hospital NHS Trust

🇬🇧

Brighton, England, United Kingdom

Radiation Safety Service, Medical Physics Department

🇬🇧

Brighton, England, United Kingdom

Histopathology Department

🇬🇧

Nottingham, England, United Kingdom

Nottingham University Hospital

🇬🇧

Nottingham, England, United Kingdom

Department of Radiology

🇬🇧

Truro, England, United Kingdom

Royal Cornwall Hospitals NHS trust

🇬🇧

Truro, England, United Kingdom

Clinical Investigation & Research Unit

🇬🇧

Brighton, Sussex, United Kingdom

ATTN-Research Pharmacist

🇺🇸

Aurora, Colorado, United States

University of Colorado Cancer Center - Anschutz Cancer Pavilion

🇺🇸

Aurora, Colorado, United States

University of Colorado Hospital, Anschutz Outpatient Pavilion

🇺🇸

Aurora, Colorado, United States

Rocky Mountain Cancer Centers

🇺🇸

Lone Tree, Colorado, United States

Florida Cancer Specialists

🇺🇸

Venice, Florida, United States

Northwestern Medical Faculty Foundation

🇺🇸

Chicago, Illinois, United States

Northwestern Memorial Hospital

🇺🇸

Chicago, Illinois, United States

The University of Chicago Medical Center,

🇺🇸

Chicago, Illinois, United States

The University of Chicago

🇺🇸

Chicago, Illinois, United States

University of Chicago Comprehensive Cancer Center at Silver Cross Hospital

🇺🇸

New Lenox, Illinois, United States

Memorial Sloan Kettering Cancer Center

🇺🇸

New York, New York, United States

Oncology Hematology Care, Inc.

🇺🇸

Fairfield, Ohio, United States

Greenville Health System

🇺🇸

Spartanburg, South Carolina, United States

Tennessee Oncology, PLLC

🇺🇸

Smyrna, Tennessee, United States

The Sarah Cannon Research Institute

🇺🇸

Nashville, Tennessee, United States

Vanderbilt Breast Center at One Hundred Oaks

🇺🇸

Nashville, Tennessee, United States

Vanderbilt Health Pharmacy One Hundred Oaks

🇺🇸

Nashville, Tennessee, United States

Texas Oncology-Longview Cancer Center

🇺🇸

Longview, Texas, United States

Texas Oncology - Tyler

🇺🇸

Tyler, Texas, United States

Virginia Cancer Institute

🇺🇸

Richmond, Virginia, United States

UZA

🇧🇪

Edegem, Antwerpen, Belgium

GZA

🇧🇪

Wilrijk, Antwerpen, Belgium

Institut Jules Bordet

🇧🇪

Brussels, Belgium

Hospital Universitario Ramon Y Cajal

🇪🇸

Madrid, Spain

Hospital Universitario 12 Octubre

🇪🇸

Madrid, Spain

Centro Integral Oncologico Clara Campal

🇪🇸

Madrid, Spain

Hospital de Madrid Norte Sanchinarro

🇪🇸

Madrid, Spain

McGill University Health Center- Cedars Cancer Center

🇨🇦

Montreal, Quebec, Canada

McGill University Health Centre - Cedars Cancer Centre

🇨🇦

Montreal, Quebec, Canada

Institute for Cancer Research

🇮🇪

Dublin, Ireland

Pharmacy Department

🇬🇧

Truro, England, United Kingdom

Radiology Department

🇬🇧

Nottingham, England, United Kingdom

St Vincent's University Hospital

🇮🇪

Dublin, Ireland

Cancer Clinical Trials Unit

🇮🇪

Dublin, Ireland

Mater Private Hospital

🇮🇪

Dublin, Ireland

IRCCS Ospedale San Raffaele

🇮🇹

Milano, Italy

U.O. Oncologia Medica, Nuovo Ospedale di Prato

🇮🇹

Prato, Italy

Divisione di Senologia Medica; Istituto Europeo di Oncologia

🇮🇹

Milano, MI, Italy

A.O.di Perugia S. Maria Della Misericoridia

🇮🇹

Perugia, PG, Italy

Azienda Ospedaliera S.Orsola Malpighi

🇮🇹

Bologna, Italy

U.O. Farmaceutica, Nuovo Ospedale di Prato

🇮🇹

Prato, Italy

© Copyright 2025. All Rights Reserved by MedPath