Safety and Efficacy Study of Sapanisertib in Combination With Exemestane or Fulvestrant in Postmenopausal Women With Estrogen Receptor Positive/Human Epidermal Growth Factor Receptor 2 Negative (ER+/HER2-) Metastatic Breast Cancer

NCT ID: NCT02049957

Last Updated: 2023-02-08

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

118 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-02-13

Study Completion Date

2018-06-29

Brief Summary

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This is a phase 1b/2 study of the safety and efficacy of sapanisertib (MLN0128) in combination with exemestane or fulvestrant therapy in women with estrogen receptor positive/human epidermal growth factor receptor 2 negative (ER+/HER2-) advanced or metastatic breast cancer who progressed on treatment with everolimus in combination with exemestane or fulvestrant.

Detailed Description

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The drug being tested in this study is called sapanisertib (MLN0128). Sapanisertib is being tested in women with estrogen receptor positive/human epidermal growth factor receptor 2 negative (ER+/HER2-) advanced or metastatic breast cancer who progressed on treatment with everolimus. This study will look at the safety and efficacy of sapanisertib when given in combination with exemestane or fulvestrant.

The study enrolled 118 patients. This study has two phases: phase 1 and phase 2. Phase 1 has 2 parts. In part 1 of phase 1, unmilled active pharmaceutical ingredient (API) capsules were administered, while in part 2, capsules based on milled API were administered.

* Phase 1 (Part 1): sapanisertib 5 mg (unmilled) + exemestane
* Phase 1 (Part 1): sapanisertib 5 mg (unmilled) + fulvestrant
* Phase 1 (Part 2): sapanisertib 3 mg (milled) + exemestane
* Phase 1 (Part 2): sapanisertib 3 mg (milled) + fulvestrant
* Phase 1 (Part 2): sapanisertib 4 mg (milled) + exemestane

In phase 2, participants were enrolled into one of 2 parallel cohorts, depending on the quality and/or duration of their prior response to everolimus in combination with either exemestane (any country) or fulvestrant (US only).

Everolimus-Resistant Cohort: patients who had progressed on treatment with everolimus in combination with either exemestane (any country) or fulvestrant (US only) without achieving an objective response (CR or PR) or after achieving stable disease for \<6 months as their best response.

Everolimus-Sensitive Cohort: participants who had progressed on treatment after achieving a CR or PR of any duration, or stable disease for ≥6 months with prior everolimus treatment in combination with either exemestane (any country) or fulvestrant (US only). Participants were to receive MLN0128 in combination with the same dose of the previously administered treatment (exemestane \[any country\] or fulvestrant \[US only\]).

This multi-center trial will be conducted worldwide. The overall time to participate in this study was 52 months. Participants made multiple visits to the clinic and were contacted by telephone every 3 months for a follow-up assessment.

Conditions

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Breast Cancer

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Phase 1 (Part 1): Sapanisertib 5 mg + Exemestane

Sapanisertib 5 mg, unmilled active pharmaceutical ingredient (API) capsule, once daily in a 28-day cycle plus exemestane 25 mg, tablets, once daily in a 28-day cycle (Up to 12 cycles).

Group Type EXPERIMENTAL

Sapanisertib

Intervention Type DRUG

Sapnisertib capsules

Exemestane

Intervention Type DRUG

Exemestane tablets.

Phase 1 (Part 1): Sapanisertib 5 mg + Fulvestrant

Sapanisertib 5 mg, unmilled API capsule, once daily in a 28-day cycle plus fulvestrant 500 mg, injection, intramuscularly (IM), once on Day 1 of each cycle (Up to 57 cycles).

Group Type EXPERIMENTAL

Sapanisertib

Intervention Type DRUG

Sapnisertib capsules

Fulvestrant

Intervention Type DRUG

Fulvestrant IM injection.

Phase 1 (Part 2): Sapanisertib 3 mg + Exemestane

Sapanisertib 3 mg, milled API capsule, once daily in a 28-day cycle plus exemestane 25 mg, tablets, once daily in a 28-day cycle (Up to 8 cycles).

Group Type EXPERIMENTAL

Sapanisertib

Intervention Type DRUG

Sapnisertib capsules

Exemestane

Intervention Type DRUG

Exemestane tablets.

Phase 1 (Part 2): Sapanisertib 3 mg + Fulvestrant

Sapanisertib 3 mg, milled API capsule, once daily in a 28-day cycle up to 14 cycles plus fulvestrant 500 mg, injection, IM, once on Day 1 of each cycle (Up to 14 cycles).

Group Type EXPERIMENTAL

Sapanisertib

Intervention Type DRUG

Sapnisertib capsules

Fulvestrant

Intervention Type DRUG

Fulvestrant IM injection.

Phase 1 (Part 2): Sapanisertib 4 mg + Exemestane

Sapanisertib 4 mg, milled API capsule, once daily, in a 28-day cycle plus exemestane 25 mg, tablets, once daily in a 28-day cycle (Up to 18 cycles).

Group Type EXPERIMENTAL

Sapanisertib

Intervention Type DRUG

Sapnisertib capsules

Exemestane

Intervention Type DRUG

Exemestane tablets.

Phase 2: Sapanisertib 4 mg + Exemestane (Everolimus Sensitive)

Sapanisertib 4 mg, milled API capsule once daily in a 28-day cycle plus exemestane 25 mg, tablets, once daily in a 28-day cycle (Up to 14 cycles) in everolimus sensitive participants.

Group Type EXPERIMENTAL

Sapanisertib

Intervention Type DRUG

Sapnisertib capsules

Exemestane

Intervention Type DRUG

Exemestane tablets.

Phase 2:Sapanisertib 4 mg+Fulvestrant (Everolimus Sensitive)

Sapanisertib 4 mg, milled API capsule once daily in a 28-day cycle plus fulvestrant 500 mg, injection IM, once on Day 1 of each cycle (Up to 17 cycles) in everolimus sensitive participants.

Group Type EXPERIMENTAL

Sapanisertib

Intervention Type DRUG

Sapnisertib capsules

Fulvestrant

Intervention Type DRUG

Fulvestrant IM injection.

Phase 2: Sapanisertib 4 mg+Exemestane (Everolimus Resistant)

Sapanisertib 4 mg, milled API capsule, once daily, in a 28-day cycle plus once daily in a 28-day cycle (Up to 12 cycles) in everolimus resistant participants.

Group Type EXPERIMENTAL

Sapanisertib

Intervention Type DRUG

Sapnisertib capsules

Exemestane

Intervention Type DRUG

Exemestane tablets.

Phase 2: Sapanisertib 4 mg+Fulvestrant (Everolimus Resistant)

Sapanisertib 4 mg, milled API capsule, once daily in a 28-day cycle plus fulvestrant 500 mg, injection IM, once on Day 1 of each cycle (Up to 9 cycles) in everolimus resistant participants.

Group Type EXPERIMENTAL

Sapanisertib

Intervention Type DRUG

Sapnisertib capsules

Fulvestrant

Intervention Type DRUG

Fulvestrant IM injection.

Interventions

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Sapanisertib

Sapnisertib capsules

Intervention Type DRUG

Fulvestrant

Fulvestrant IM injection.

Intervention Type DRUG

Exemestane

Exemestane tablets.

Intervention Type DRUG

Other Intervention Names

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MLN0128

Eligibility Criteria

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Inclusion Criteria

Phase 1b and Phase 2

1. Advanced or metastatic breast cancer.
2. Histological or cytological confirmation of ER+ status (defined as \> 1% positive tumor cells), and histological or cytological confirmation of HER2-negative (HER2-) status by local laboratory testing using criteria in the American Society of Oncology (ASCO)/College of American Pathologists (CAP) Clinical Practice Guideline update.
3. Female patients 18 years of age or older who are postmenopausal for at least 1 year before the Screening visit, where menopause is defined by: Age ≥ 55 years and 1 year or more of amenorrhea. Surgical menopause with bilateral oophorectomy

Age \< 55 years and 1 year or more of amenorrhea, with an estradiol assay \< 20 pg/mL

Note: Ovarian radiation or treatment with a luteinizing hormone-releasing hormone agonist (goserelin acetate or leuprolide acetate) is not permitted for induction of ovarian suppression.
4. Have a history of brain metastasis are eligible for the study provided that all the following criteria are met:

Brain metastases which have been treated
* No evidence of disease progression for ≥ 3 months or hemorrhage after treatment
* Off-treatment with dexamethasone for 4 weeks before administration of the first dose of MLN0128
* No ongoing requirement for dexamethasone or anti-epileptic drugs
5. Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.
6. Clinical laboratory values as specified below within 4 weeks before the first dose of MLN0128:

* Bone marrow reserve consistent with absolute neutrophil count (ANC) ≥ 1.5 x 10\^9/L; platelet count ≥ 100 x 10\^9/L; hemoglobin ≥ 9 g/dL
* Total bilirubin ≤ 1.5 x the upper limit of the normal range (ULN), aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN (≤ 5 x ULN if liver metastases are present)
* Creatinine clearance ≥ 50 mL/min based either on Cockcroft-Gault estimate or based on a 12- or 24-hour urine collection
* Fasting serum glucose ≤ 130 mg/dL and fasting triglycerides ≤ 300 mg/dL
7. Left ventricular ejection fraction (LVEF) within 5 absolute percentage points of institutional standard of normal as measured by echocardiogram (ECHO) or multiple gated acquisition scan (MUGA) within 4 weeks before the first dose of MLN0128 (ie, if the institutional standard of normal is 50%, LVEF may be as low as 45% to be eligible for the study).
8. Able to provide paraffin blocks or a minimum of 10 unstained slides of available archival tumor tissues (paraffin blocks are preferred). If archival tumor tissue is not available, a tumor biopsy may be performed before the patient begins treatment with MLN0128. If fewer than 10 slides are available or the tumor content/area requirements are not met, study eligibility will be determined upon discussion with the sponsor.
9. Ability to swallow oral medications, willingness to perform mucositis prophylaxis, and suitable venous access for the study-required blood sampling.
10. Voluntary written consent must be given before the performance of any study related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to future medical care.

11. Patients may have SD or disease progression during their most recent treatment with exemestane or fulvestrant, or everolimus in combination with either exemestane (any country) or fulvestrant (US only). Exemestane or fulvestrant in combination with MLN0128 can also be initiated as a new line of therapy.

12. Measurable disease defined as follows:

* At least 1 extra-osseous lesion that can be accurately measured in at least 1 dimension. The lesion must measure ≥ 20 mm with conventional imaging techniques or ≥ 10 mm with spiral computed tomography (CT) or magnetic resonance imaging (MRI), or
* Bone lesions (lytic or mixed \[lytic plus sclerotic\]) in the absence of measurable disease as defined above
13. Patients must have had disease progression during treatment with everolimus in combination with either exemestane (any country) or fulvestrant (US only) (duration of treatment ≥ 4 weeks) and must have tolerated everolimus treatment in combination with exemestane (any country) or fulvestrant (US only) adequately according to the treating physician's judgment. Everolimus in combination with exemestane or fulvestrant is not required to be the most recent treatment before enrollment, but progression on the most recent anticancer therapy is required for enrollment.

Exclusion Criteria

Phase 1b and Phase 2

1. Prior anticancer therapy or other investigational therapy within 2 weeks before administration of the first dose of MLN0128 (except for exemestane or fulvestrant, which should be continued). Treatment with everolimus must be discontinued 2 weeks before administration of the first dose of MLN0128.
2. Chronic concomitant therapy with bisphosphonates or denosumab for the prevention of bone metastases. Concomitant treatment with bisphosphonates or denosumab is permitted for treatment of osteoporosis or management of existing bone metastases if initiated at least 4 weeks before administration of the first dose of MLN0128.
3. Initiation of treatment with hematopoietic growth factors, transfusions of blood and blood products, or systemic corticosteroids (either IV or oral steroids, excluding inhalers) within 1 week before administration of the first dose of MLN0128 (patients already receiving erythropoietin on a chronic basis for ≥ 4 weeks are eligible).
4. Previous treatment with dual PI3K/mTOR inhibitors or TORC1/2 inhibitors.
5. Manifestations of malabsorption due to prior gastrointestinal (GI) surgery, GI disease, or for an unknown reason that may alter the absorption of MLN0128.
7. Other clinically significant co-morbidities, such as uncontrolled pulmonary disease, active central nervous system disease, active infection, or any other condition that could compromise participation of the patient in the study.
8. Known human immunodeficiency virus infection.
9. History of any of the following within the last 6 months before administration of the first dose of MLN0128:

* Ischemic myocardial event, including angina requiring therapy and artery revascularization procedures
* Ischemic cerebrovascular event, including transient ischemic attack and artery revascularization procedures
* Requirement for inotropic support (excluding digoxin) or serious (uncontrolled) cardiac arrhythmia (including atrial flutter/fibrillation, ventricular fibrillation, or ventricular tachycardia)
* Placement of a pacemaker for control of rhythm
* New York Heart Association Class III or IV heart failure
* Pulmonary embolism
10. Significant active cardiovascular or pulmonary disease before administration of the first dose of MLN0128, including:

* Uncontrolled hypertension (ie, systolic blood pressure \> 180 mm Hg; diastolic blood pressure \> 95 mm Hg)
* Pulmonary hypertension
* Uncontrolled asthma or oxygen saturation \< 90% by arterial blood gas analysis or pulse oximetry on room air
* Significant valvular disease; severe regurgitation or stenosis by imaging independent of symptom control with medical intervention; or history of valve replacement
* Medically significant (symptomatic) bradycardia
* History of arrhythmia requiring an implantable cardiac defibrillator
* Baseline prolongation of the rate-corrected QT interval (QTc; eg, repeated demonstration of QTc interval \> 480 ms, or history of congenital long QT syndrome, or torsades de pointes)
11. Diagnosed or treated for another malignancy within 2 years before administration of the first dose of MLN0128 or previously diagnosed with another malignancy and have any evidence of residual disease. Patients with nonmelanoma skin cancer or carcinoma in situ of any type are not excluded if they have undergone complete resection.

12. More than 3 prior chemotherapy regimens for locally advanced or metastatic disease.

13. More than 1 prior chemotherapy regimen for locally advanced or metastatic disease.
Minimum Eligible Age

18 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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Calithera Biosciences, Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Monitor

Role: STUDY_DIRECTOR

Millennium Pharmaceuticals, Inc.

Locations

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Los Angeles Hematology

Los Angeles, California, United States

Site Status

University of California at San Francisco (PARENT)

San Francisco, California, United States

Site Status

Santa Barbara Hematology Oncology Medical Group, Inc.

Santa Barbara, California, United States

Site Status

University of Colorado Cancer Center

Aurora, Colorado, United States

Site Status

Rocky Mountain Cancer Centers, LLP

Lakewood, Colorado, United States

Site Status

Mount Sinai Medical Center

Miami Beach, Florida, United States

Site Status

Florida Cancer Research Institute

Plantation, Florida, United States

Site Status

University of Kansas Medical Center Research Institute, Inc.

Westwood, Kansas, United States

Site Status

Holy Cross Hospital

Silver Spring, Maryland, United States

Site Status

Henry Ford Medical Center

Novi, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Roswell Park Cancer Institute

Buffalo, New York, United States

Site Status

Herbert Irving Comprehensive Cancer Center

New York, New York, United States

Site Status

Weill Cornell Medical College New York Presbyterian Hospital

New York, New York, United States

Site Status

Eastchester Center for Cancer Care / BRANY

The Bronx, New York, United States

Site Status

University of Cincinnati Physicians Company, LLC

Cincinnati, Ohio, United States

Site Status

University Hospitals of Cleveland

Cleveland, Ohio, United States

Site Status

Erlanger Medical Center

Chattanooga, Tennessee, United States

Site Status

Texas Oncology, P.A. - Beaumont

Beaumont, Texas, United States

Site Status

Texas Oncology, P.A.

Dallas, Texas, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Millennium Oncology

Houston, Texas, United States

Site Status

Texas Health Physicians Group

Plano, Texas, United States

Site Status

Cancer Care Network of South Texas - SAT&BC

San Antonio, Texas, United States

Site Status

Texas Oncology, P.A. - Tyler

Tyler, Texas, United States

Site Status

Virginia Oncology Associates - Hampton

Chesapeake, Virginia, United States

Site Status

Oncology and Hematology Assoc. of SW VA, Inc.

Salem, Virginia, United States

Site Status

West Virginia University

Morgantown, West Virginia, United States

Site Status

UZ Antwerpen

Antwerp, , Belgium

Site Status

Institut Jules Bordet

Brussels, , Belgium

Site Status

Universitair Ziekenhuis Brussel

Brussels, , Belgium

Site Status

GHdC Notre Dame

Charleroi, , Belgium

Site Status

Centre Hospitalier de l'Ardenne

Libramont, , Belgium

Site Status

GZA Ziekenhuizen - Campus Sint-Augustinus

Wilrijk, , Belgium

Site Status

Centre Francois Baclesse

Caen, Calvados, France

Site Status

Centre Catherine de Sienne

Nantes, Loire Atlantique, France

Site Status

Clinique Victor Hugo - Centre Jean Bernard

Le Mans, Sarthe, France

Site Status

Institut Sainte Catherine

Avignon, Vaculuse, France

Site Status

Countries

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United States Belgium France

References

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Lim B, Potter DA, Salkeni MA, Silverman P, Haddad TC, Forget F, Awada A, Canon JL, Danso M, Lortholary A, Bourgeois H, Tan-Chiu E, Vincent S, Bahamon B, Galinsky KJ, Patel C, Neuwirth R, Leonard EJ, Diamond JR. Sapanisertib Plus Exemestane or Fulvestrant in Women with Hormone Receptor-Positive/HER2-Negative Advanced or Metastatic Breast Cancer. Clin Cancer Res. 2021 Jun 15;27(12):3329-3338. doi: 10.1158/1078-0432.CCR-20-4131. Epub 2021 Apr 5.

Reference Type DERIVED
PMID: 33820779 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2014-001921-34

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1195-3894

Identifier Type: REGISTRY

Identifier Source: secondary_id

C31001

Identifier Type: -

Identifier Source: org_study_id

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