Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant in Men and Postmenopausal Women With Advanced Breast Cancer Which Progressed on or After Aromatase Inhibitor Treatment.

NCT ID: NCT02437318

Last Updated: 2025-02-13

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

572 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-07-23

Study Completion Date

2023-06-09

Brief Summary

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To determine whether treatment with alpelisib plus fulvestrant prolonged progression-free survival (PFS) compared to fulvestrant and placebo in men and postmenopausal women with hormone receptor positive (HR+), human epidermal growth factor receptor-2 (HER2)-negative advanced breast cancer, who received prior treatment with an aromatase Inhibitor (AI) either as (neo)adjuvant or for advanced disease.

Detailed Description

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This was a randomized, double-blind, placebo-controlled, international multicenter Phase III study that evaluated the efficacy and safety of treatment with alpelisib plus fulvestrant versus placebo plus fulvestrant in men and postmenopausal women with HR-positive, HER2-negative advanced breast cancer which had progressed on or after AI treatment.

Subjects were allocated to either the PIK3CA mutant or PIK3CA non-mutant cohort, based on central testing of hotspot-mutations in tumor tissue. Subjects with unknown results were not eligible. Within each cohort, subjects were randomized in a 1:1 ratio to receive either alpelisib 300 mg orally once daily (q.d.), in combination with fulvestrant 500 mg intramuscular (i.m.) on Days 1 and 15 of Cycle 1 and Day 1 of a 28-day cycle thereafter, or placebo daily in combination with fulvestrant 500 mg following the same treatment regimen.

Subjects were treated until disease progression, unacceptable toxicity, death, or discontinuation from the study treatment for any other reason. All subjects who discontinued study treatment were followed for safety, until 30 days after last study treatment administration, except in the case of death, loss to follow-up, or withdrawal of consent.

Subjects who discontinued study treatment for reasons other than disease progression or withdrawal of consent, were followed until disease progression, death, withdrawal of consent, loss to follow-up, or subject/guardian decision (post-treatment efficacy follow-up).

Finally, all subjects were followed for survival after discontinuation of study treatment and tumor evaluations until the subject's death, loss to follow-up, or withdrawal of consent for survival follow-up (post-treatment survival follow-up)

Conditions

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Breast Cancer

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Fulvestrant + alpelisib

Subjects treated with alpelisib (300 mg; oral; once daily) in combination with fulvestrant (500 mg; intramuscular injection on Day 1 and Day 15 of Cycle 1, and then Day 1 of each subsequent 28-day cycle)

Group Type EXPERIMENTAL

Fulvestrant

Intervention Type DRUG

500 mg of fulvestrant administered via intramuscular injection on Day 1 and Day 15 of Cycle 1, and then Day 1 of each subsequent 28-day cycle

Alpelisib

Intervention Type DRUG

300 mg of alpelisib tablets for oral use administered once daily

Fulvestrant + placebo

Subjects were treated with placebo (300 mg; oral; once daily) in combination with fulvestrant (500 mg; intramuscular injection on Day 1 and Day 15 of Cycle 1, and then Day 1 of each subsequent 28-day cycle)

Group Type PLACEBO_COMPARATOR

Fulvestrant

Intervention Type DRUG

500 mg of fulvestrant administered via intramuscular injection on Day 1 and Day 15 of Cycle 1, and then Day 1 of each subsequent 28-day cycle

Placebo

Intervention Type DRUG

300 mg of placebo tablets for oral use administered once daily

Interventions

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Fulvestrant

500 mg of fulvestrant administered via intramuscular injection on Day 1 and Day 15 of Cycle 1, and then Day 1 of each subsequent 28-day cycle

Intervention Type DRUG

Alpelisib

300 mg of alpelisib tablets for oral use administered once daily

Intervention Type DRUG

Placebo

300 mg of placebo tablets for oral use administered once daily

Intervention Type DRUG

Other Intervention Names

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BYL719

Eligibility Criteria

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Inclusion Criteria

* If female, the patient was postmenopausal.
* The patient had identified PIK3CA status.
* Patients could be:
* Relapsed with documented evidence of progression while on (neo)adjuvant endocrine therapy or within 12 months from completion of (neo)adjuvant endocrine therapy with no treatment for metastatic disease.
* Relapsed with documented evidence of progression more than 12 months from completion of (neo)adjuvant endocrine therapy and then subsequently progressed with documented evidence of progression while on or after only one line of endocrine therapy for metastatic disease.
* Newly diagnosed with advanced breast cancer, then relapsed with documented evidence of progression while on or after only one line of endocrine therapy.
* The patient had recurrence or progression of the disease during or after AI therapy (i.e., letrozole, anastrozole, exemestane).
* The patient had a histologically and/or cytologically confirmed diagnosis of estrogen-receptor positive breast cancer by a local laboratory and had HER2 negative breast cancer.
* The patient had either measurable disease per RECIST 1.1 criteria or at least one predominantly lytic bone lesion present.
* The patient had adequate bone marrow function.

Exclusion Criteria

* The patient had symptomatic visceral disease or any disease burden that made the patient ineligible for endocrine therapy per the investigator's best judgment.
* The patient had received prior treatment with chemotherapy (except for neoadjuvant/adjuvant chemotherapy), fulvestrant, any PI3K, mTOR, or AKT inhibitor (pre-treatment with CDK4/6 inhibitors was allowed).
* The patient had inflammatory breast cancer at screening.
* Patients had Child pugh score B or C.
* Patients had an established diagnosis of diabetes mellitus type I or uncontrolled type II.
* The patient had Eastern Cooperative Oncology Group (ECOG) performance status 2 or more.
* The patient had CNS involvement unless he/she was at least 4 weeks from prior therapy completion to starting the study treatment and had a stable CNS tumor at the time of screening and was not receiving steroids and/or enzyme-inducing antiepileptic medications for brain metastases.
* The patient had participated in a prior investigational study within 30 days prior to enrollment or within 5 half-lives of the investigational product, whichever was longer.
* The patient had a history of acute pancreatitis within 1 year of screening or a past medical history of chronic pancreatitis.
* The patient relapsed with documented evidence of progression more than 12 months from completion of (neo)adjuvant endocrine therapy with no treatment for metastatic disease.
Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Szekszárd, , Hungary

Site Status

Ironwood Cancer and Research Centers

Chandler, Arizona, United States

Site Status

Mayo Clinic Arizona

Scottsdale, Arizona, United States

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Highlands Oncology Group

Fayetteville, Arkansas, United States

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Beverly Hills Cancer Center

Beverly Hills, California, United States

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City of Hope National Medical Center

Duarte, California, United States

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Scripps Green Hospital

La Jolla, California, United States

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Kaiser Permanent Southern Californi

San Diego, California, United States

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UCSF

San Francisco, California, United States

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Florida Cancer Specialists

Fort Myers, Florida, United States

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Florida Cancer Specialists-North

St. Petersburg, Florida, United States

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Rush University Medical Center

Chicago, Illinois, United States

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NorthShore University Health System

Evanston, Illinois, United States

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Edward Cancer Center

Naperville, Illinois, United States

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Fort Wayne Medical Oncology Hematology Inc

Fort Wayne, Indiana, United States

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St Francis Health Comprehensive Cancer Center

Topeka, Kansas, United States

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Mercy Medical Center

Baltimore, Maryland, United States

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Massachusetts General Hospital

Boston, Massachusetts, United States

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Lahey Clinic

Burlington, Massachusetts, United States

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Detroit Clinical Research Center

Owosso, Michigan, United States

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St Lukes Cancer Institute

Kansas City, Missouri, United States

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St Vincent Frontier Cancer Center

Billings, Montana, United States

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Rutgers Cancer Institute of New Jersey

New Brunswick, New Jersey, United States

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University Hospitals of Cleveland Seidman Cancer Center

Cleveland, Ohio, United States

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Good Samaritan Regional Medical Center

Corvallis, Oregon, United States

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Lancaster General Hospital

Lancaster, Pennsylvania, United States

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Prisma Health Upstate

Greenville, South Carolina, United States

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Avera Cancer

Sioux Falls, South Dakota, United States

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SCRI Oncology Partners

Nashville, Tennessee, United States

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Texas Oncology PA Dallas Presbyterian Hospital

Dallas, Texas, United States

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El Paso Texas Oncology

El Paso, Texas, United States

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Mays Cancer Ctr Uthsa Mdacc

San Antonio, Texas, United States

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Texas Oncology Northeast Texas

Tyler, Texas, United States

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Virginia Cancer Specialists

Fairfax, Virginia, United States

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Wenatchee Valley Medical Center

Wenatchee, Washington, United States

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Novartis Investigative Site

Berazategui, Buenos Aires, Argentina

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CABA, Buenos Aires, Argentina

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Rio Negro, Viedma, Argentina

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La Rioja, , Argentina

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Wahroonga, New South Wales, Australia

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Wooloongabba, Queensland, Australia

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Elizabeth Vale, South Australia, Australia

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Melbourne, Victoria, Australia

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Linz, , Austria

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Vienna, , Austria

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Sint-Niklaas, Oost Vlaanderen, Belgium

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Brussels, , Belgium

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Brussels, , Belgium

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Edegem, , Belgium

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Libramont, , Belgium

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Ottignies, , Belgium

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Verviers, , Belgium

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Natal, Rio Grande do Norte, Brazil

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Lajeado, Rio Grande do Sul, Brazil

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São Paulo, São Paulo, Brazil

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São José do Rio Preto, , Brazil

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Plovdiv, , Bulgaria

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Sofia, , Bulgaria

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Varna, , Bulgaria

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Temuco, Región de la Araucanía, Chile

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Santiago, , Chile

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Viña del Mar, , Chile

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Prague, Czech Republic, Czechia

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Zlín, Czech Republic, Czechia

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Hradec Králové, CZE, Czechia

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Lübeck, Schleswig-Holstein, Germany

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Recklinghausen, , Germany

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Ulm, , Germany

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Velbert, , Germany

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Thessaloniki, GR, Greece

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Pokfulam, , Hong Kong

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Nyíregyháza, , Hungary

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Haifa, , Israel

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Petah Tikva, , Israel

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Ramat Gan, , Israel

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Tel Aviv, , Israel

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Ancona, AN, Italy

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Chieti, CH, Italy

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Meldola, FC, Italy

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Milan, MI, Italy

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Padua, PD, Italy

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Aviano, PN, Italy

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Rionero in Vulture, PZ, Italy

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Roma, RM, Italy

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Savona, SV, Italy

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Torino, TO, Italy

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Negrar, VR, Italy

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Sassari, , Italy

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Nagoya, Aichi-ken, Japan

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Matsuyama, Ehime, Japan

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Maebashi, Gunma, Japan

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Sapporo, Hokkaido, Japan

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Akashi, Hyōgo, Japan

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Isehara, Kanagawa, Japan

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Osaka, Osaka, Japan

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Osaka, Osaka, Japan

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Kitaadachi-gun, Saitama, Japan

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Chuo Ku, Tokyo, Japan

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Minato Ku, Tokyo, Japan

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Kagoshima, , Japan

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Kumamoto, , Japan

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Beirut, , Lebanon

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El Achrafiyé, , Lebanon

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Saida, , Lebanon

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Monterrey, Nuevo León, Mexico

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Venray, CE, Netherlands

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Terneuzen, , Netherlands

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San Borja, Lima region, Peru

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Surquillo, Lima region, Peru

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Lima, , Peru

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Floreşti, Cluj, Romania

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Craiova, Dolj, Romania

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Iași, , Romania

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Arkhangelsk, , Russia

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Ryazan, , Russia

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Saint Petersburg, , Russia

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Bundang Gu, Gyeonggi-do, South Korea

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Gyeonggi-do, Korea, South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seville, Andalusia, Spain

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Palma de Mallorca, Balearic Islands, Spain

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Jerez de la Frontera, Cadiz, Spain

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Badalona, Catalonia, Spain

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Barcelona, Catalonia, Spain

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Badajoz, Extremadura, Spain

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Cáceres, Extremadura, Spain

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Santiago de Compostela, Galicia, Spain

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Pozuelo de Alarcón, Madrid, Spain

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El Palmar, Murcia, Spain

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San Cristóbal de La Laguna, Santa Cruz De Tenerife, Spain

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Alicante, Valencia, Spain

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Castellon, Valencia, Spain

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Valencia, Valencia, Spain

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Madrid, , Spain

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Madrid, , Spain

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Madrid, , Spain

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Madrid, , Spain

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Madrid, , Spain

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Gävle, , Sweden

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Örebro, , Sweden

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Västerås, , Sweden

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Taipei, , Taiwan

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Taipei, , Taiwan

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Bangkok, , Thailand

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Bangkok, , Thailand

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Leicester, , United Kingdom

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London, , United Kingdom

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Plymouth, , United Kingdom

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Countries

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United States Argentina Australia Austria Belgium Brazil Bulgaria Canada Chile Czechia France Germany Greece Hong Kong Hungary India Israel Italy Japan Lebanon Mexico Netherlands Peru Romania Russia South Korea Spain Sweden Taiwan Thailand United Kingdom

References

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Borrego MR, Lu YS, Reyes-Cosmelli F, Park YH, Yamashita T, Chiu J, Airoldi M, Turner N, Fein L, Ghaznawi F, Singh J, Pantoja K, Schnell C, Akdere M, Chia S. SGLT2 inhibition improves PI3Kalpha inhibitor-induced hyperglycemia: findings from preclinical animal models and from patients in the BYLieve and SOLAR-1 trials. Breast Cancer Res Treat. 2024 Nov;208(1):111-121. doi: 10.1007/s10549-024-07405-8. Epub 2024 Aug 23.

Reference Type DERIVED
PMID: 39177931 (View on PubMed)

Rodon J, Demanse D, Rugo HS, Burris HA, Simo R, Farooki A, Wellons MF, Andre F, Hu H, Vuina D, Quadt C, Juric D. A risk analysis of alpelisib-induced hyperglycemia in patients with advanced solid tumors and breast cancer. Breast Cancer Res. 2024 Mar 4;26(1):36. doi: 10.1186/s13058-024-01773-1.

Reference Type DERIVED
PMID: 38439079 (View on PubMed)

Ciruelos EM, Rugo HS, Mayer IA, Levy C, Forget F, Delgado Mingorance JI, Safra T, Masuda N, Park YH, Juric D, Conte P, Campone M, Loibl S, Iwata H, Zhou X, Park J, Ridolfi A, Lorenzo I, Andre F. Patient-Reported Outcomes in Patients With PIK3CA-Mutated Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor 2-Negative Advanced Breast Cancer From SOLAR-1. J Clin Oncol. 2021 Jun 20;39(18):2005-2015. doi: 10.1200/JCO.20.01139. Epub 2021 Mar 29.

Reference Type DERIVED
PMID: 33780274 (View on PubMed)

Andre F, Ciruelos EM, Juric D, Loibl S, Campone M, Mayer IA, Rubovszky G, Yamashita T, Kaufman B, Lu YS, Inoue K, Papai Z, Takahashi M, Ghaznawi F, Mills D, Kaper M, Miller M, Conte PF, Iwata H, Rugo HS. Alpelisib plus fulvestrant for PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor-2-negative advanced breast cancer: final overall survival results from SOLAR-1. Ann Oncol. 2021 Feb;32(2):208-217. doi: 10.1016/j.annonc.2020.11.011. Epub 2020 Nov 25.

Reference Type DERIVED
PMID: 33246021 (View on PubMed)

Rugo HS, Andre F, Yamashita T, Cerda H, Toledano I, Stemmer SM, Jurado JC, Juric D, Mayer I, Ciruelos EM, Iwata H, Conte P, Campone M, Wilke C, Mills D, Lteif A, Miller M, Gaudenzi F, Loibl S. Time course and management of key adverse events during the randomized phase III SOLAR-1 study of PI3K inhibitor alpelisib plus fulvestrant in patients with HR-positive advanced breast cancer. Ann Oncol. 2020 Aug;31(8):1001-1010. doi: 10.1016/j.annonc.2020.05.001. Epub 2020 May 13.

Reference Type DERIVED
PMID: 32416251 (View on PubMed)

Andre F, Ciruelos E, Rubovszky G, Campone M, Loibl S, Rugo HS, Iwata H, Conte P, Mayer IA, Kaufman B, Yamashita T, Lu YS, Inoue K, Takahashi M, Papai Z, Longin AS, Mills D, Wilke C, Hirawat S, Juric D; SOLAR-1 Study Group. Alpelisib for PIK3CA-Mutated, Hormone Receptor-Positive Advanced Breast Cancer. N Engl J Med. 2019 May 16;380(20):1929-1940. doi: 10.1056/NEJMoa1813904.

Reference Type DERIVED
PMID: 31091374 (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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2015-000340-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CBYL719C2301

Identifier Type: -

Identifier Source: org_study_id

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