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Efficacy Evaluation of Enobosarm Monotherapy in Treatment of AR+/ER+/HER2- Metastatic Breast Cancer

Phase 3
Terminated
Conditions
Metastatic Breast Cancer
Interventions
Registration Number
NCT04869943
Lead Sponsor
Veru Inc.
Brief Summary

To demonstrate the efficacy of enobosarmin the treatment of androgen receptor positive (AR+) and estrogen receptor positive (ER+) metastatic breast cancer (MBC) as measured by radiographic progression free survival (rPFS).

Detailed Description

This study is a multicenter, randomized, open-label, two treatment arm, efficacy and safety study, comprised of two sections, the main study and the post-study extension period. Subjects will be randomized to the two treatment arms in a 1:1 fashion (into the main study), with the opportunity for subjects initially randomized to the Control Treatment Group to cross over to receive the investigational treatment in the post-study extension period.

The primary efficacy endpoint of the study will be the median rPFS. Subjects will continue study treatment until disease progression confirmed by blinded independent central reader (BICR) is observed. A safety follow up visit will occur approximately 30 days after last dose of study drug. Thereafter, survival follow up will be completed monthly for one year. Survival follow up may be completed by phone or records review. After one year, survival follow up will be completed every 90 days.

After radiographic progression (by RECIST 1.1) is confirmed by blinded independent central reader (BICR) and have received approval from Medical Monitor, subjects in the Control Treatment Group may be crossed over to receive enobosarm treatment (9mg per day). Treatment will continue in this population (Enobosarm Post-study Group (EPG)) until radiographic progression (by RECIST 1.1), confirmed by BICR, is observed. The efficacy database for this crossover group will be completely separate from the main portion of the study and the data will be analyzed separately.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
52
Inclusion Criteria
  • Provide informed consent

  • Be able to communicate effectively with the study personnel

  • Aged ≥18 years

  • For Female Subjects

    • Menopausal status
  • Be postmenopausal as defined by the National Comprehensive Cancer Network as either:

    • Age ≥55 years and one year or more of amenorrhea
    • Age <55 years and one year or more of amenorrhea, with an estradiol assay <20 pg/mL
    • Age <55 years and surgical menopause with bilateral oophorectomy
  • Be premenopausal or perimenopausal on ovarian suppression with LHRH agonist for at least 4 months, with an estradiol assay <20 pg/mL and a negative urine pregnancy test.

  • If subject is of child bearing potential, the subject must agree to use acceptable methods of contraception:

    • If female study participant could become pregnant, use acceptable methods of contraception from the time of the first administration of study medication until 6 months following administration of the last dose of study medication. Acceptable methods of contraception are as follows: Condom with spermicidal foam/gel/film/cream/suppository [i.e., barrier method of contraception], surgical sterilization of male partner (vasectomy with documentation of azoospermia) and a barrier method {condom used with spermicidal foam/gel/film/cream/suppository}
    • If female study participant has undergone documented tubal ligation (female sterilization), a barrier method (condom used with spermicidal foam/gel/film/cream/suppository) should also be used
    • If female study participant has undergone documented placement of an intrauterine device (IUD) or intrauterine system (IUS), a barrier method (condom with spermicidal foam/gel/film/cream/suppository) should also be used
  • For Male Subjects

    • Subject must agree to use acceptable methods of contraception:
  • If the study subject's partner could become pregnant, use acceptable methods of contraception from the time of the first administration of study medication until 6 months following administration of the last dose of study medication. Acceptable methods of contraception are as follows: Condom with spermicidal foam/gel/ film/cream/suppository [i.e., barrier method of contraception], surgical sterilization (vasectomy with documentation of azoospermia) and a barrier method {condom used with spermicidal foam/gel/film/cream/suppository}, the female partner uses oral contraceptives (combination estrogen/progesterone pills), injectable progesterone or subdermal implants and a barrier method (condom used with spermicidal foam/gel/film/cream/suppository)

  • If female partner of a study subject has undergone documented tubal ligation (female sterilization), a barrier method (condom used with spermicidal foam/gel/film/cream/suppository) should also be used

  • If female partner of a study subject has undergone documented placement of an intrauterine device (IUD) or intrauterine system (IUS), a barrier method (condom with spermicidal foam/gel/film/cream/suppository) should also be used

  • Eastern Cooperative Oncology Group (ECOG) performance status of ≤2

  • Documented evidence of ER+/HER2- metastatic breast cancer

  • Measurable disease is required as per RECIST 1.1 (NOTE: Bone only metastatic disease is acceptable but requires a measurable component

  • Have androgen receptor nuclei staining ≥40% as assessed by central laboratory

  • Received at least 2 prior lines of treatment in MBC setting which must have included both an AI (monotherapy or combination) and fulvestrant (monotherapy or combination); at least one must have been given in combination with a CDK 4/6 inhibitor.

  • Previously responded (without disease progression for at least 6 months) to one of the following treatments: fulvestrant monotherapy or fulvestrant plus CDK 4/6 inhibitor or nonsteroidal aromatase inhibitor monotherapy or nonsteroidal aromatase inhibitor plus CDK 4/6 inhibitor for metastatic breast cancer.

  • Subject is willing to comply with the requirements of the protocol through the end of the study

Exclusion Criteria
  • Known hypersensitivity or allergy to enobosarm
  • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2.5 X upper limit of normal (ULN) or total bilirubin >ULN (an elevated total bilirubin up to 1.5 X ULN attributed to a previously confirmed diagnosis of Gilbert's disease is acceptable if all other eligibility criteria are met). In patients with documented metastases to the liver, the limits for inclusion are ALT or AST >5.0 X ULN or total bilirubin >1.5 X ULN.
  • Patients with biliary catheter.
  • Creatinine clearance < 30 mL/min as measured using the Cockcoft Gault formula (patients with mild and moderate renal failure are not excluded from participation in this study)
  • Previously received >1 course of systemic chemotherapy (not including immunotherapies or targeted therapies) for the treatment of metastatic breast cancer.

Note: Subjects may have received 1 course of chemotherapy in the adjuvant or neoadjuvant setting would not count as a line of therapy.

  • Subjects with radiographic evidence of central nervous system (CNS) metastases as assessed by CT or MRI that are not well-controlled (symptomatic or requiring control with continuous corticosteroid therapy [e.g., dexamethasone]) Note: Subjects with CNS metastases are permitted to participate in the study if the CNS metastases are medically well-controlled and stable for at least 30 days after receiving local therapy (irradiation, surgery, etc.)

  • Radiotherapy within 14 days prior to randomization except in case of localized radiotherapy for analgesic purpose or for lytic lesions at risk of fracture, which can then be completed within 7 days prior to randomization. Subjects must have recovered from radiotherapy toxicities prior to randomization

  • Any comorbid disease or condition (medical or surgical) which might compromise the hematologic, cardiovascular, endocrine, pulmonary, severe renal impairment, gastrointestinal, hepatic, or central nervous system; or other conditions that may interfere with the absorption, distribution, metabolism or excretion of study drug, or would place the subject at increased risk

  • Treatment with any investigational product within < 4 half-lives for each individual investigational product OR within 30 days prior to randomization

  • Major surgery within 30 days prior to randomization

  • Treatment with testosterone, methyltestosterone, oxandrolone (Oxandrin®), oxymetholone, danazol, fluoxymesterone (Halotestin®), testosterone-like agents (such as dehydroepiandrosterone, androstenedione, and other androgenic compounds, including herbals), or antiandrogens (enzalutamide, abiraterone, bicalutamide, apalutamide, or darolutamide). Previous therapy with testosterone and testosterone-like agents is acceptable with a 30-day washout (if previous testosterone therapy was long term depot within the past 6 months, the site should contact the Medical Monitor) or any other androgenic agent.

  • Treatment with any of the following hormone replacement therapies for metastatic breast cancer. Prior use in the adjuvant or neoadjuvant setting is allowed if the treatment is, discontinued greater than 30 days prior to randomization

    • Estrogens
    • Megesterol acetate
    • Testosterone
  • All other concurrent anticancer treatments (including, but not limited to, all SERMs unless randomized to the Control Treatment Group with a SERM as the control treatment, AIs unless randomized to Control Treatment Group (exemestane or exemestane plus everolimus) with the AI containing treatment as the control treatment, and all CDK 4/6 inhibitors)

  • An abnormal ECG result which, based on the investigator's clinical judgment, would place the subject at increased risk

  • Has a known additional, invasive, malignancy that is progressing or required active treatment in the last 5 years [note: subjects with basal cell carcinoma of the skin, squamous cell carcinoma of the skin, ductal breast carcinoma in situ, bladder cancer (superficial treated), or cervical carcinoma in situ that have undergone potentially curative therapy are not excluded]

  • Pregnant, lactating, or breastfeeding, or intending to become pregnant during the study or within 60 days after the final dose of study treatment

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Control Treatment GroupExemestaneSubjects in the Control Treatment Group will receive an ER targeted therapy limited to exemestane monotherapy, exemestane plus everolimus or selective estrogen receptor modulator (SERM) approved for the treatment of breast cancer and is part of the standard of care at the clinical study site. The decision of which comparator treatment will be used will be made prior to randomization. After radiographic progression, subjects randomized to the Control Treatment Group may be crossed over to receive enobosarm 9mg.
Enobosarm Treatment GroupEnobosarmSubjects in the Enobosarm Treatment Group will receive enobosarm 9mg each day by mouth until disease progression or an unacceptable adverse event is observed. The total duration of the study for a subject in the study from screening to follow-up visit is not standardized and will be different for each subject.
Primary Outcome Measures
NameTimeMethod
To demonstrate the efficacy of Enobosarm in the treatment of androgen receptor positive (AR+) and estrogen receptor positive (ER+) metastatic breast cancer (MBC) as measured by radiographic progression free survival (rPFS).Day 120

The primary endpoint for the study is the median radiographic progression free survival (rPFS) in the Enobosarm Treatment Group compared to the Control Treatment Group.

Progression will be defined based on RECIST 1.1.

Secondary Outcome Measures
NameTimeMethod
Objective Response Rate (ORR)Day 180

Objective Response Rate (ORR), proportion of subjects with a best tumor response of ORR (PR or CR) on study

Trial Locations

Locations (53)

Banner Health/ Banner MD Anderson Cancer Center

🇺🇸

Gilbert, Arizona, United States

Texas Oncology Sammons Cancer Center

🇺🇸

Dallas, Texas, United States

University of Miami- Sylvester Comprehensive Cancer Center

🇺🇸

Miami, Florida, United States

Miami Cancer Institute

🇺🇸

Miami, Florida, United States

Comprehensive Cancer Centers of Nevada

🇺🇸

Las Vegas, Nevada, United States

The Lindner Center for Research and Education at the Christ Hospital

🇺🇸

Cincinnati, Ohio, United States

Baptist Clinical Research Institute

🇺🇸

Nashville, Tennessee, United States

Rocky Mountain Cancer Centers

🇺🇸

Denver, Colorado, United States

Inspira Medical Center

🇺🇸

Vineland, New Jersey, United States

California Research Institute (CRI)

🇺🇸

Los Angeles, California, United States

Magee-Women's Hospital

🇺🇸

Pittsburgh, Pennsylvania, United States

Texas Oncology - Tyler

🇺🇸

Tyler, Texas, United States

Marin Cancer Care, Inc.

🇺🇸

Greenbrae, California, United States

Hospital Ruber Internacional

🇪🇸

Madrid, Spain

Blessing Corporate Services

🇺🇸

Quincy, Illinois, United States

MultiCare Institute for Research and Innovation

🇺🇸

Spokane, Washington, United States

Hospital Universitari Dexeus

🇪🇸

Barcelona, Spain

Hospital Universitari Vall d'Hebron - Vall d'Hebron Institut d'Oncologia (VHIO)

🇪🇸

Barcelona, Spain

Oncology and Hematology Associates of Southwest Virginia, Inc.

🇺🇸

Roanoke, Virginia, United States

A Coruña University Hospital

🇪🇸

A Coruña, Spain

Hospital Clínico Universitario de Valencia (CHUV)

🇪🇸

Valencia, Spain

Hospital Universitario HM Sanchinarro

🇪🇸

Madrid, Spain

Hospital Universitario 12 de Octubre (H12O)

🇪🇸

Madrid, Spain

Dana-Farber Cancer Institute Breast Oncology

🇺🇸

Boston, Massachusetts, United States

MBCCOP - LSU Health Sciences Center

🇺🇸

Shreveport, Louisiana, United States

Tennessee Cancer Specialists

🇺🇸

Knoxville, Tennessee, United States

The Oncology Insitute of Hope and Innovation

🇺🇸

Glendale, California, United States

Providence Medical Group

🇺🇸

Santa Rosa, California, United States

University of California San Francisco Comprehensive Cancer Center

🇺🇸

San Francisco, California, United States

Woodlands Medical Specialists, PA

🇺🇸

Pensacola, Florida, United States

University Cancer & Blood Center

🇺🇸

Athens, Georgia, United States

Virginia Oncology Associates

🇺🇸

Norfolk, Virginia, United States

Virginia Cancer Specialists

🇺🇸

Fairfax, Virginia, United States

"Oddział Onkologii Klinicznej i Chemioterapii Europejskie Centrum Zdrowia Otwock"

🇵🇱

Otwock, Poland

Cancer Care Northwest

🇺🇸

Spokane, Washington, United States

Institut Catala d'Oncologia (ICO)

🇪🇸

Barcelona, Spain

Hospital Universitari Arnau de Vilanova de Lleida

🇪🇸

Lleida, Spain

State institution "V.T. Zaycev Institute of general and urgent surgery of National academy medical sciences of Ukraine"

🇺🇦

Kharkiv, Ukraine

Morton Plant Hospital/ BayCare Health System, Inc

🇺🇸

Clearwater, Florida, United States

Ironwood Cancer and Research Centers

🇺🇸

Chandler, Arizona, United States

Astera Cancer Care

🇺🇸

East Brunswick, New Jersey, United States

Khmelnytsky Regional Antitumor Center, Department of Breast, Skin, Soft Tissues and Bones Tumorsa

🇺🇦

Khmelnytskyi, Ukraine

Miami Cancer Institute, Plantation MCIP

🇺🇸

Plantation, Florida, United States

Tidelands Health

🇺🇸

Murrells Inlet, South Carolina, United States

Specjalistyczny Szpital Onkologiczny NU-MED

🇵🇱

Maków Mazowiecki, Poland

Centrum Onkologii Ziemi Lubelskiej im. św. Jana z Dukli

🇵🇱

Lublin, Poland

Odessa Regional Oncological Dispensary

🇺🇦

Odessa, Ukraine

Inspira Medical Center Mullica Hill

🇺🇸

Mullica Hill, New Jersey, United States

Wojewódzka Przychodnia Onkologiczna, Wojewódzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Łodzi

🇵🇱

Łódź, Poland

Municipal Institution "Dnipropetrovsk City Multi-field Clinical Hospital #4", Dnepropetrovsk state m

🇺🇦

Dnepropetrovsk, Ukraine

Klinika Nowotworów Piersi i Chirurgii Rekonstrukcyjnej, Narodowy Instytut Onkologii im. Marii Skłodowskiej-Curie Państwowy Instytut Badawczy

🇵🇱

Warsaw, Poland

Instytut Centrum Zdrowia Matki Polki

🇵🇱

Łódź, Poland

Kyiv City Clinical Oncology Center

🇺🇦

Kyiv, Ukraine

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