NCT02007512|CompletedPhase 2Results

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

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

Pfizer ClinicalTrials.gov

Compare

3 other identifiers

MDV3100-122013-002717-35C3431008

Study Type

interventional

Target

247

Locations

7 countries

Sites

135

Timeline

RegisteredDec 2013

StartedDec 2013

CompletionAug 2024

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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

247

participants targeted

Target at P75+ for phase_2 breast-cancer

Timeline

Completed

Started Dec 2013

Longer than P75 for phase_2 breast-cancer

Geographic Reach

7 countries

135 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

10.7 years study duration

First Submitted

Initial submission to the registry

December 3, 2013

Completed

7 days until next milestone

First Posted

Study publicly available on registry

December 10, 2013

Completed

6 days until next milestone

Study Start

First participant enrolled

December 16, 2013

Completed

2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 23, 2016

Completed

1.4 years until next milestone

Results Posted

Study results publicly available

February 6, 2018

Completed

6.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

August 23, 2024

Completed

Last Updated

September 2, 2025

Status Verified

August 1, 2025

Enrollment Period

2.8 years

First QC Date

December 3, 2013

Results QC Date

September 21, 2017

Last Update Submit

August 11, 2025

Conditions

Breast Cancer

Keywords

advanced breast cancerenzalutamideMDV3100Estrogen receptor positive (ER +)Progesterone receptor positive (PgR +)HER-2 normal

Outcome Measures

Primary Outcomes (2)

Progression Free Survival (PFS): Intent-to-Treat (ITT) Population By Interactive Web Recognition System (IWRS)
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.
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)
Progression Free Survival (PFS): Diagnostic Positive (DX+) Population By Interactive Web Recognition System (IWRS)
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.
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)

Secondary Outcomes (9)

Clinical Benefit Rate-24 (CBR-24)
From randomization up to 3 years
Best Objective Response Rate
From randomization until CR or PR, whichever occurred first (up to 3 years)
Duration of Objective Response
From 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)
Time to Response
From 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 Progression
From randomization until PD or last tumor assessment without PD before new antitumor treatment initiation, whichever occurred first (up to 3 years)
+4 more secondary outcomes

Other Outcomes (9)

Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) Quality-of-Life Core Questionnaire (QLQ-C30) at Weeks 5, 9, 17, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121 and 133: Global Health/Quality of Life
Baseline; Weeks 5, 9, 17, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121 and 133
Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) Breast Cancer Module (QLQ-BR23) at Weeks 5, 9, 17, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121 and 133
Baseline; Weeks 5, 9, 17, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121 and 133
Number of Participants With Positive Androgen Receptor (AR) Expression by Immunohistochemistry (IHC)
Day 1, 29, 57, 113 and 169
+6 more other outcomes

Study Arms (2)

Enzalutamide & exemestane

EXPERIMENTAL

Enzalutamide 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.

Drug: EnzalutamideDrug: exemestane

Placebo & exemestane

ACTIVE COMPARATOR

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.

Drug: exemestaneDrug: Placebo (for enzalutamide)

Interventions

EnzalutamideDRUG

160 mg/day administered as four 40mg soft gelatin capsules by mouth once daily with or without food.

Also known as: MDV3100, XTANDI

Enzalutamide & exemestane

exemestaneDRUG

25mg (overencapsulated to match 50mg dose during the blinded portion of the study and one 25mg tablet after unblinding) by mouth once daily after food.

Also known as: Aromasin

Placebo & exemestane

Placebo (for enzalutamide)DRUG

Sugar pill manufactured to mimic enzalutamide administered as four soft gelatin capsules by mouth once daily with or without food.

Placebo & exemestane

Eligibility Criteria

Age18 Years+

Sexfemale(Gender-based eligibility)

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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;

You may not qualify if:

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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Pfizerlead
Astellas Pharma Inccollaborator
Medivation, Inc.collaborator

Study Sites (135)

ATTN-Research Pharmacist

Aurora, Colorado, 80045, United States

Location

University of Colorado Cancer Center - Anschutz Cancer Pavilion

Aurora, Colorado, 80045, United States

Location

University of Colorado Hospital, Anschutz Outpatient Pavilion

Aurora, Colorado, 80045, United States

Location

Rocky Mountain Cancer Centers

Colorado Springs, Colorado, 80907, United States

Location

Rocky Mountain Cancer Centers

Lakewood, Colorado, 80228, United States

Location

Rocky Mountain Cancer Centers

Lone Tree, Colorado, 80124, United States

Location

Florida Cancer Specialists

Altamonte Springs, Florida, 32701, United States

Location

Florida Cancer Specialists

Bonita Springs, Florida, 34135, United States

Location

Florida Cancer Specialists

Bradenton, Florida, 34209, United States

Location

Florida Cancer Specialists

Brandon, Florida, 33511, United States

Location

Florida Cancer Specialists

Cape Coral, Florida, 33909, United States

Location

Florida Cancer Specialists

Clearwater, Florida, 33761, United States

Location

Florida Cancer Specialists

Englewood, Florida, 34223, United States

Location

Florida Cancer Specialists

Fort Myers, Florida, 33901, United States

Location

Florida Cancer Specialists

Fort Myers, Florida, 33905, United States

Location

Florida Cancer Specialists

Fort Myers, Florida, 33908, United States

Location

Florida Cancer Specialists

Gainesville, Florida, 32605, United States

Location

Florida Cancer Specialists

Hudson, Florida, 34667, United States

Location

Florida Cancer Specialists

Largo, Florida, 33770, United States

Location

Florida Cancer Specialists

Naples, Florida, 34102, United States

Location

Florida Cancer Specialists

New Port Richey, Florida, 34655, United States

Location

Florida Cancer Specialists

Orange City, Florida, 32763, United States

Location

Florida Cancer Specialists

Orlando, Florida, 32806, United States

Location

Florida Cancer Specialists

Port Charlotte, Florida, 33980, United States

Location

Florida Cancer Specialists

Sarasota, Florida, 34232, United States

Location

Florida Cancer Specialists

Sarasota, Florida, 34236, United States

Location

Florida Cancer Specialists

Spring Hill, Florida, 34608, United States

Location

Florida Cancer Specialists

St. Petersburg, Florida, 33705, United States

Location

Florida Cancer Specialists

Tampa, Florida, 33607, United States

Location

Florida Cancer Specialists

Tavares, Florida, 32778, United States

Location

Florida Cancer Specialists

Venice, Florida, 34285, United States

Location

Florida Cancer Specialists

Venice, Florida, 34292, United States

Location

Northwestern Medical Faculty Foundation

Chicago, Illinois, 60611, United States

Location

Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

Location

The University of Chicago Medical Center,

Chicago, Illinois, 60637, United States

Location

The University of Chicago

Chicago, Illinois, 60637, United States

Location

University of Chicago Comprehensive Cancer Center at Silver Cross Hospital

New Lenox, Illinois, 60451, United States

Location

Indiana University Health Hospital

Indianapolis, Indiana, 46202, United States

Location

Indiana University Health Melvin and Bren Simon Cancer Center

Indianapolis, Indiana, 46202, United States

Location

Investigational Drug Services

Indianapolis, Indiana, 46202, United States

Location

Sidney and Lois Eskenazi Hospital

Indianapolis, Indiana, 46202, United States

Location

Springmill Medical Clinic

Indianapolis, Indiana, 46290, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Minnesota Oncology Hematology, P.A

Minneapolis, Minnesota, 55404, United States

Location

Abbott Northwestern Hospital

Minneapolis, Minnesota, 55407, United States

Location

Allina Health System DBA Virginia Piper Cancer Institute

Minneapolis, Minnesota, 55407, United States

Location

Dr.Michaela Tsai

Minneapolis, Minnesota, 55407, United States

Location

The West Clinic, P.C.

Corinth, Mississippi, 38834, United States

Location

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

Southaven, Mississippi, 38671, United States

Location

Siteman Cancer Center

City of Saint Peters, Missouri, 63376, United States

Location

Barnes-Jewish Hospital

St Louis, Missouri, 63110, United States

Location

Washington University Infusion Center Pharmacy

St Louis, Missouri, 63110, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Siteman Cancer Center-South County

St Louis, Missouri, 63129, United States

Location

Siteman Cancer Center- West County

St Louis, Missouri, 63141, United States

Location

Hematology Oncology Associates of Northern NJ

Morristown, New Jersey, 07962, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location