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

Trial tests alpelisib plus fulvestrant for advanced breast cancer with PIK3CA mutation.

NCT: NCT05038735 · Status: ACTIVE NOT RECRUITING · Phase: Phase 3 · Sponsor: Novartis Pharmaceuticals · Started: 2021-12-17 · Est. Completion: 2027-02-26

Plain English Summary

Study to Assess the Efficacy and Safety of Alpelisib Plus Fulvestrant in Participants With HR-positive (HR+), HER2-negative, Advanced Breast Cancer After Treatment With a CDK4/6 Inhibitor and an Aromatase Inhibitor. is a Phase 3 clinical trial sponsored by Novartis Pharmaceuticals studying Breast Cancer. This trial tests if a combination of alpelisib and fulvestrant is more effective than placebo plus fulvestrant for certain types of advanced breast cancer. It is for men and postmenopausal women whose breast cancer is HR-positive, HER2-negative, has a PIK3CA mutation, and has progressed after prior treatments. Participants will receive either alpelisib plus fulvestrant or a placebo plus fulvestrant, assigned randomly. This involves taking pills and receiving injections. Alternative treatments include other chemotherapy or targeted therapies, depending on the patient's specific situation and prior treatments. The trial aims to enroll 234 participants.

Official Summary

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.

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults aged 18 or older who have been diagnosed with HR-positive, HER2-negative advanced breast cancer. Patients whose cancer has a specific PIK3CA mutation and has worsened after treatment with an aromatase inhibitor and a CDK4/6 inhibitor. Individuals with at least one measurable tumor lesion. Women must be postmenopausal. Men are also eligible. Patients who have received prior treatment with fulvestrant or certain other targeted therapies may not be eligible. This trial is studying Breast Cancer, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcome measures how long patients live without their cancer getting worse, which is crucial for understanding how well the new treatment combination controls the disease. The specific primary outcome measures are: Progression-free survival (PFS) based on BIRC assessments and using RECIST v1.1 criteria (From randomization to date of the first documented progression or death due to any cause, assessed up to a maximum duration of 60 months.). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial addresses a significant unmet need for patients with advanced HR-positive, HER2-negative breast cancer with a PIK3CA mutation who have exhausted standard treatment options. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Breast Cancer, where improved treatment options are needed.

Investor Insight

This Phase III trial, sponsored by Novartis, investigates a targeted therapy for a specific breast cancer subtype, indicating a focus on precision medicine with potential for significant market impact Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.

Is This Trial Right for Me?

Ask your doctor if your cancer has a PIK3CA mutation and if you have progressed on prior treatments. Understand that you will be randomly assigned to receive either the study drugs or a placebo, and this assignment is blinded. Participation involves regular clinic visits for assessments, blood tests, and receiving the study medication (pills and injections). The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

Interventions

Primary Outcomes

Secondary Outcomes

Full Eligibility Criteria

Key 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.
* 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) is permitted.
* The presence of PIK3CA mutation(s) determined in tumor tissue prior to 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.
* If female, then the participant must be in postmenopausal status.

Key Exclusion Criteria:

* Participant with symptomatic visceral disease or any disease burden 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 (SERD), any Phosphatidylinositol-3-Kinase (PI3K), mammalian Target of Rapamycin (mTOR) or Protein Kinase B (AKT) inhibitor.

Other Inclusion and Exclusion Criteria do apply

Trial Locations

Frequently Asked Questions

What is clinical trial NCT05038735?

NCT05038735 is a Phase 3 INTERVENTIONAL study titled "Study to Assess the Efficacy and Safety of Alpelisib Plus Fulvestrant in Participants With HR-positive (HR+), HER2-negative, Advanced Breast Cancer After Treatment With a CDK4/6 Inhibitor and an Aromatase Inhibitor.." It is currently active, not recruiting and is sponsored by Novartis Pharmaceuticals. The trial targets enrollment of 234 participants.

What conditions does NCT05038735 study?

This trial investigates treatments for Breast Cancer. The primary condition under study is Breast Cancer.

What treatments are being tested in NCT05038735?

The interventions being studied include: Alpelisib (DRUG), Fulvestrant (DRUG), Alpelisib-matching placebo (DRUG). Alpelisib (tablets) administered at 300mg orally once daily on a continuous dosing schedule starting on Cycle 1 Day 1 in a 28 day cycle.

What does Phase 3 mean for NCT05038735?

Phase 3 trials are large-scale studies involving 300-3,000+ patients that compare the new treatment against existing standard treatments. Positive Phase 3 results are typically required for FDA approval.

What is the current status of NCT05038735?

This trial is currently "Active, Not Recruiting." It started on 2021-12-17. The estimated completion date is 2027-02-26.

Who is sponsoring NCT05038735?

NCT05038735 is sponsored by Novartis Pharmaceuticals. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT05038735?

The trial aims to enroll 234 participants. The trial status is active, not recruiting.

How is NCT05038735 designed?

This is a interventional study, uses randomized allocation, follows a crossover design, employs triple masking. Masking means some participants and/or investigators do not know which treatment group a participant is in, which helps reduce bias.

What are the primary outcomes being measured in NCT05038735?

The primary outcome measures are: Progression-free survival (PFS) based on BIRC assessments and using RECIST v1.1 criteria (From randomization to date of the first documented progression or death due to any cause, assessed up to a maximum duration of 60 months.). These are the main endpoints researchers use to determine whether the treatment is effective.

Where is NCT05038735 being conducted?

This trial is being conducted at 20 sites, including Sint-Niklaas, Oost Vlaanderen; Brussels; Ghent; Leuven and 16 more sites (Belgium, Bulgaria, Canada).

Where can I find official information about NCT05038735?

The official record for NCT05038735 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT05038735. This government database provides the most up-to-date and detailed information about the trial.

What is NCT05038735 testing in simple terms?

This trial tests if a combination of alpelisib and fulvestrant is more effective than placebo plus fulvestrant for certain types of advanced breast cancer. It is for men and postmenopausal women whose breast cancer is HR-positive, HER2-negative, has a PIK3CA mutation, and has progressed after prior treatments.

Why is this trial significant?

This trial addresses a significant unmet need for patients with advanced HR-positive, HER2-negative breast cancer with a PIK3CA mutation who have exhausted standard treatment options. As a Phase 3 trial, positive results could lead directly to regulatory approval and new treatment options for patients.

What are the potential risks of participating in NCT05038735?

Common side effects may include high blood sugar, diarrhea, nausea, and rash. Serious side effects can occur, including severe diarrhea, hyperglycemia (high blood sugar), and potential effects on blood cell counts. The study involves a placebo, meaning some participants will not receive the active study drug, which could affect outcomes. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT05038735?

Ask your doctor if your cancer has a PIK3CA mutation and if you have progressed on prior treatments. Understand that you will be randomly assigned to receive either the study drugs or a placebo, and this assignment is blinded. Participation involves regular clinic visits for assessments, blood tests, and receiving the study medication (pills and injections). Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT05038735 signal from an investment perspective?

This Phase III trial, sponsored by Novartis, investigates a targeted therapy for a specific breast cancer subtype, indicating a focus on precision medicine with potential for significant market impact This is a Phase 3 trial, which is the final pivotal stage before potential regulatory submission.

What happens if the treatment in this trial doesn't work?

Participants will receive either alpelisib plus fulvestrant or a placebo plus fulvestrant, assigned randomly. This involves taking pills and receiving injections. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.

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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.