Last Update: Nov 14, 2024
EPIK-B5: A Phase III, Randomized, Double-blind, Placebo-controlled Study of Alpelisib in Combination With Fulvestrant for Men and Postmenopausal Women With HR-positive, HER2-negative Advanced Breast Cancer With a PIK3CA Mutation, Who Progressed on or After Aromatase Inhibitor and a CDK4/6 Inhibitor
ClinicalTrials.gov Identifier:
Novartis Reference Number:CBYL719C2303
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

The purpose of this study is to complement Study CBYL719C2301 (SOLAR-1) and obtain more
comprehensive data on the efficacy and safety of alpelisib (BYL719) in combination with
fulvestrant compared with placebo plus fulvestrant in men or postmenopausal women with
HR-positive, HER2-negative advanced breast cancer with a PIK3CA mutation who progressed
or relapsed on or after treatment with an AI plus a CDK4/6 inhibitor. This is a Phase III, randomized, double-blind, placebo-controlled, international,
multi-center trial. Approximately 234 men and postmenopausal women will be randomized to
either alpelisib plus fulvestrant or alpelisib-matching placebo plus fulvestrant.
Randomization will follow a 1:1 randomization ratio and be stratified by presence of lung
and/or liver metastases (yes vs. no) and setting at last prior CDK4/6 inhibitor therapy
(adjuvant vs metastatic).

Study treatment with alpelisib plus fulvestrant or alpelisib-matching placebo plus
fulvestrant will be initiated on Cycle 1 Day 1, and will continue until disease
progression per RECIST v1.1 as per BIRC assessment, start of new antineoplastic therapy,
death, lost to follow-up, or withdrawal of consent. A cycle is defined as 28 days.

Participants randomized to the alpelisib-matching placebo plus fulvestrant arm who have
disease progression per RECIST v1.1 as assessed by BIRC will have the option to crossover
to be treated with alpelisib plus fulvestrant.

Unblinding a single participant at a site will be permitted after disease progression
confirmed by BIRC after discussion with the Novartis team to determine eligibility for
cross-over to treatment with alpelisib plus fulvestrant.

Breast Cancer
Phase3
Recruiting
234
Nov 29, 2021
Dec 29, 2027
All
18 Years - (Adult, Older Adult)

Interventions

Drug

Alpelisib

Alpelisib (tablets) administered at 300mg orally once daily on a continuous dosing schedule starting on Cycle 1 Day 1 in a 28 day cycle.
Drug

Alpelisib-matching placebo

Alpelisib-matching placebo (tablets) administered orally once daily on a continuous dosing schedule starting on Cycle 1 Day 1 in a 28 day cycle.
Drug

Fulvestrant

Fulvestrant (prefilled syringe) 500mg administered intramuscularly at Cycle 1 Day 1 and 15 and then at Day 1 of each subsequent cycle (each cycle is 28 days).

Eligibility Criteria

Inclusion Criteria:

- Participant is an adult ≥ 18 years old at the time of informed consent and has
signed informed consent before any trial related activities and according to local
guidelines.

- If female, then the participant must be in postmenopausal status. Postmenopausal
status is defined either by: prior bilateral oophorectomy, age ≥60 or age <60 and
amenorrheic for 12 or more months in the absence of chemotherapy, tamoxifen,
toremifene, or ovarian suppression and follicle-stimulating hormone (FSH) and
estradiol in the postmenopausal range per local normal range.

- 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 (Fluorescent in situ hybridization (FISH), Chromogenic in situ
hybridization (CISH), or Silver-enhanced in situ hybridization (SISH)) test is
required by local laboratory testing.

- Participant has at least one measurable lesion as per RECIST v1.1 criteria as
assessed by Investigator (a lesion at a previously irradiated site may only be
counted as a target lesion if there is clear sign of progression since the
irradiation).

- Participant has recurrence or progression of disease during or after combined AI
(i.e. letrozole, anastrozole, exemestane) and CDK4/6 inhibitor therapy. The combined
AI and CDK4/6 inhibitor therapy does not need to be the latest treatment regimen
(including adjuvant setting).

- Participant has received ≤2 prior lines of systemic therapies overall in the
metastatic setting, of which a maximum of 1 line of prior treatment with
chemotherapy (except for neoadjuvant/ adjuvant chemotherapy).

- Participant must show the presence of a PIK3CA mutation(s) determined in tumor
tissue prior ro enrollment either by a Novartis designated laboratory or in tumor
tissue or plasma ctDNA by a local laboratory using a Food and Drug Administration
(FDA)-approved PIK3CA Companion Diagnostics (CDx) test for alpelisib or the CE-IVD
QIAGEN Therascreen® PIK3CA RGQ PCR test.

Exclusion Criteria:

- Participant with symptomatic visceral disease that makes the participant ineligible
for endocrine therapy (ET) per the investigator's best judgment.

- Participant who relapsed with documented evidence of progression more than 12 months
from completion of (neo)adjuvant endocrine/endocrine-based therapy with no treatment
for metastatic disease

- Participant has received prior treatment with fulvestrant, any oral selective
estrogen receptor degrader (SERDs), any Phosphatidylinositol-3-Kinase (PI3K),
mammalian Target of Rapamycin (mTOR) or Protein Kinase B (AKT) inhibitor

Other Inclusion and Exclusion Criteria do apply

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