Study Assessing the Efficacy and Safety of Treatment With Alpelisib Plus Fulvestrant in Japanese Men and Postmenopausal Women With Advanced Breast Cancer

  • End date
    Feb 1, 2027
  • participants needed
  • sponsor
    Novartis Pharmaceuticals
Updated on 26 April 2021
measurable disease
human epidermal growth factor
epidermal growth factor
aromatase inhibitor
her2-negative breast cancer


The purpose of this study is to assess the safety and efficacy of alpelisib plus fulvestrant in men and postmenopausal women with hormone receptor (HR) positive, human epidermal growth factor 2 (HER2)-negative, advanced breast cancer harboring a PIK3CA mutation in Japan, whose disease has progressed on or after aromatase inhibitor (AI) treatment regardless of prior CDK4/6 inhibitor use.


This is a Phase II open-label, 2-Part, multi-center study in Japan. The study will be conducted in two parts: Part 1 (Cohort 1) includes participants regardless of prior CDK4/6 inhibitor use and is designed to determine the recommended dose (RD), evaluate the tolerability and safety of alpelisib in combination with fulvestrant. Part 2 consists of 2 cohorts (the CDK4/6 inhibitor naive participants are in Cohort 2 and the CDK4/6 inhibitor pre-treated participants are in Cohort 3) which are designed to assess the efficacy and safety of alpelisib in combination with fulvestrant, will start once the RD of alpelisib is established.

Participants will be treated until disease progression, unacceptable toxicity, death or discontinuation from the study treatment for any other reason and will be followed for survival regardless of treatment discontinuation reason (except if consent is withdrawn or participant is lost to follow up).

Condition Advanced Breast Cancer
Treatment fulvestrant, Alpelisib
Clinical Study IdentifierNCT04524000
SponsorNovartis Pharmaceuticals
Last Modified on26 April 2021


Yes No Not Sure

Inclusion Criteria

Japanese man or postmenopausal woman
Participant has adequate tumor tissue for the analysis of PIK3CA mutational status by a Novartis designated laboratory
Participant has identified PIK3CA mutation (as determined by a Novartis designated laboratory)
Participant has a histologically and/or cytologically confirmed diagnosis of ER+ and/or PgR+ breast cancer by local laboratory
Participant has HER2-negative breast cancer defined as a negative in situ hybridization test or an IHC status of 0, 1+ or 2+. If IHC is 2+, a negative in situ hybridization (FISH, CISH or SISH) test is required by local laboratory testing
Participant has measurable disease, i.e., at least one measurable lesion as per RECIST 1.1
Participant has advanced breast cancer
Participant has ECOG performance status 0 or 1

Exclusion Criteria

Participant with symptomatic visceral disease or any disease burden that makes the participant ineligible for endocrine therapy per the investigator's best judgment
Participant has received prior treatment
with chemotherapy (except for neoadjuvant/ adjuvant chemotherapy), fulvestrant, any PI3K, mTOR or AKT inhibitor for Cohort 1 and 3
with chemotherapy (except for neoadjuvant/ adjuvant chemotherapy), fulvestrant, any PI3K, mTOR, AKT inhibitor or CDK 4/6 inhibitor for Cohort 2
Participant has a known hypersensitivity to alpelisib or fulvestrant, or to any of the excipients of alpelisib or fulvestrant
Participant with inflammatory breast cancer at screening
Participant is concurrently using other anti-cancer therapy
Participant has had surgery within 14 days prior to starting study drug or has not recovered from major side effects
Participant with an established diagnosis at screening of diabetes mellitus type I or not controlled type II (based on FPG and HbA1c)
Participant has currently documented pneumonitis /interstitial lung disease
History of acute pancreatitis within 1 year of screening or past medical history of chronic pancreatitis
Participant with unresolved osteonecrosis of the jaw
Participant has a history of severe cutaneous reactions
Other protocol-defined inclusion/exclusion criteria may apply
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