A Phase II Open-label, 2-Part, Multi-center Study of BYL719 (Alpelisib) in Combination With Fulvestrant for Men and Postmenopausal Women With PIK3CA Mutation Hormone Receptor (HR) Positive, HER2-negative, Advanced Breast Cancer Which Progressed on or After Aromatase Inhibitor (AI) Treatment in Japan
Trial of Alpelisib plus Fulvestrant for Advanced Breast Cancer in Japan
Plain English Summary
Study Assessing the Efficacy and Safety of Treatment With Alpelisib Plus Fulvestrant in Japanese Men and Postmenopausal Women With Advanced Breast Cancer is a Phase 2 clinical trial sponsored by Novartis Pharmaceuticals studying Advanced Breast Cancer. This trial tests a combination of two drugs, alpelisib and fulvestrant, to see how well they work and how safe they are. It is for men and postmenopausal women in Japan with advanced breast cancer that has a specific genetic mutation (PIK3CA) and has not responded to prior treatment. Participants will receive the study drugs, and their health will be closely monitored through regular visits and tests. Alternative treatments may include other hormone therapies, chemotherapy, or targeted therapies depending on the patient's specific situation and prior treatments. The trial aims to enroll 24 participants.
Official Summary
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.
Who Can Participate
Here is what you need to know about eligibility for this trial. Men and postmenopausal women in Japan diagnosed with advanced breast cancer. Patients must have a confirmed PIK3CA gene mutation in their tumor and HER2-negative cancer. The cancer must have progressed after treatment with aromatase inhibitors, and patients should have measurable disease. Individuals with symptomatic visceral disease, certain prior treatments (like PI3K/mTOR/AKT inhibitors or CDK4/6 inhibitors in specific cohorts), or known hypersensitivity to the study drugs cannot participate. This trial is studying Advanced Breast Cancer, so participants generally need a confirmed diagnosis.
What They're Measuring
The primary outcome measures will determine the highest safe dose of the drug combination and how often the cancer shrinks or stops growing in patients who haven't previously received CDK4/6 inhibitor The specific primary outcome measures are: [Part 1] The incidence of Dose Limiting Toxicities (DLTs) of alpelisib in combination with fulvestrant (From Cycle 1 Day 1 to Cycle 2 Day 28 (Cycle = 28 days)); [Part 2] Overall Response Rate (ORR) in CDK4/6 inhibitor naive participants (Up to approximately 36 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 2, which tests whether the treatment actually works against the target condition. Phase 2 trials involve 100-300 patients and continue to monitor safety while evaluating effectiveness. This phase often tests different dosages to find the optimal amount. About 33% of Phase 2 drugs advance to Phase 3. If successful, the treatment will move to large-scale Phase 3 trials needed for FDA approval.
Why This Trial Matters
This trial addresses a need for more effective treatments for advanced breast cancer in Japanese patients with specific genetic mutations, particularly after initial therapies have stopped working. Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets Advanced Breast Cancer, where improved treatment options are needed.
Investor Insight
This trial focuses on a specific genetic subtype of breast cancer, a growing area of targeted therapy, suggesting potential for a niche market if successful, though competition in advanced breast canc Phase 2 trials have approximately a 15-20% chance of eventually gaining FDA approval.
Is This Trial Right for Me?
Ask your doctor about the specific genetic tests needed to confirm the PIK3CA mutation and HER2 status. Understand the potential side effects of alpelisib and fulvestrant, and how they will be managed during the trial. Be prepared for regular clinic visits for drug administration, blood tests, and imaging scans to monitor your cancer and overall health. The trial is being conducted at 15 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
- Study Type: INTERVENTIONAL
- Allocation: NON_RANDOMIZED
- Model: PARALLEL
- Masking: NONE
- Enrollment: 24 participants
Interventions
- DRUG: Alpelisib — \[Part 1\] Alpelisib administered at 200 mg (DL 1), 250 mg (DL 2) or 300mg (DL 3) orally once daily on a continuous dosing schedule starting on Cycle 1 Day 1 in a 28 day cycle. If DL 1 is tolerated, the alpelisib doses of 250 mg will be investigated. If DL 2 is tolerated, the alpelisib doses of 300 mg will be investigated. \[Part 2\] In the Part 2, participants will be enrolled into Cohort 2 (CDK4/6 inhibitor naive) and Cohort 3 (CDK4/6 inhibitor pre-treated) in parallel and alpelisib will be
- DRUG: Fulvestrant — Fulvestrant is administered at a dose of 500 mg intramuscular on Cycle 1 Day 1, Day 15, and Day 1 of every cycle thereafter (where a cycle is 28 days).
Primary Outcomes
- [Part 1] The incidence of Dose Limiting Toxicities (DLTs) of alpelisib in combination with fulvestrant (From Cycle 1 Day 1 to Cycle 2 Day 28 (Cycle = 28 days))
- [Part 2] Overall Response Rate (ORR) in CDK4/6 inhibitor naive participants (Up to approximately 36 months)
Secondary Outcomes
- [Part 1] Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) (Up to approximately 37 months)
- [Part 1] Number of participants with dose adjustments (Up to approximately 37 months)
- [Part 1] Dose intensity for alpelisib and fulvestrant (Up to approximately 37 months)
- [Part 1] Duration of exposure for alpelisib and fulvestrant (Up to approximately 37 months)
- [Part 1] Plasma concentrations of alpelisib in combination with fulvestrant (Cycle 1 Day 8 (pre-dose), 15 (pre-dose, post-dose 1 hour, 3 hours) and Day 1 of Cycles 2, 4, 6, 8 (pre-dose) (Cycle = 28 days))
Full Eligibility Criteria
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
Trial Locations
- Novartis Investigative Site, Nagoya, Aichi-ken, Japan
- Novartis Investigative Site, Nagoya, Aichi-ken, Japan
- Novartis Investigative Site, Nagoya, Aichi-ken, Japan
- Novartis Investigative Site, Kashiwa, Chiba, Japan
- Novartis Investigative Site, Isehara, Kanagawa, Japan
- Novartis Investigative Site, Yokohama, Kanagawa, Japan
- Novartis Investigative Site, Sendai, Miyagi, Japan
- Novartis Investigative Site, Osaka, Osaka, Japan
- Novartis Investigative Site, Osaka, Osaka, Japan
- Novartis Investigative Site, Suita, Osaka, Japan
- ...and 5 more locations
Frequently Asked Questions
What is clinical trial NCT04524000?
NCT04524000 is a Phase 2 INTERVENTIONAL study titled "Study Assessing the Efficacy and Safety of Treatment With Alpelisib Plus Fulvestrant in Japanese Men and Postmenopausal Women With Advanced Breast Cancer." It is currently active, not recruiting and is sponsored by Novartis Pharmaceuticals. The trial targets enrollment of 24 participants.
What conditions does NCT04524000 study?
This trial investigates treatments for Advanced Breast Cancer. The primary condition under study is Advanced Breast Cancer.
What treatments are being tested in NCT04524000?
The interventions being studied include: Alpelisib (DRUG), Fulvestrant (DRUG). \[Part 1\] Alpelisib administered at 200 mg (DL 1), 250 mg (DL 2) or 300mg (DL 3) orally once daily on a continuous dosing schedule starting on Cycle 1 Day 1 in a 28 day cycle. If DL 1 is tolerated, the alpelisib doses of 250 mg will be investigated. If DL 2 is tolerated, the alpelisib doses of 300 mg will be investigated. \[Part 2\] In the Part 2, participants will be enrolled into Cohort 2 (CDK4/6 inhibitor naive) and Cohort 3 (CDK4/6 inhibitor pre-treated) in parallel and alpelisib will be
What does Phase 2 mean for NCT04524000?
Phase 2 trials test whether the treatment works for the intended condition. They involve 100-300 patients and continue to evaluate safety while measuring effectiveness.
What is the current status of NCT04524000?
This trial is currently "Active, Not Recruiting." It started on 2021-01-08. The estimated completion date is 2027-02-27.
Who is sponsoring NCT04524000?
NCT04524000 is sponsored by Novartis Pharmaceuticals. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT04524000?
The trial aims to enroll 24 participants. The trial status is active, not recruiting.
How is NCT04524000 designed?
This is a interventional study, uses non_randomized allocation, follows a parallel design, employs none masking.
What are the primary outcomes being measured in NCT04524000?
The primary outcome measures are: [Part 1] The incidence of Dose Limiting Toxicities (DLTs) of alpelisib in combination with fulvestrant (From Cycle 1 Day 1 to Cycle 2 Day 28 (Cycle = 28 days)); [Part 2] Overall Response Rate (ORR) in CDK4/6 inhibitor naive participants (Up to approximately 36 months). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT04524000 being conducted?
This trial is being conducted at 15 sites, including Nagoya, Aichi-ken; Kashiwa, Chiba; Isehara, Kanagawa; Yokohama, Kanagawa and 11 more sites (Japan).
Where can I find official information about NCT04524000?
The official record for NCT04524000 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT04524000. This government database provides the most up-to-date and detailed information about the trial.
What is NCT04524000 testing in simple terms?
This trial tests a combination of two drugs, alpelisib and fulvestrant, to see how well they work and how safe they are. It is for men and postmenopausal women in Japan with advanced breast cancer that has a specific genetic mutation (PIK3CA) and has not responded to prior treatment.
Why is this trial significant?
This trial addresses a need for more effective treatments for advanced breast cancer in Japanese patients with specific genetic mutations, particularly after initial therapies have stopped working.
What are the potential risks of participating in NCT04524000?
Common side effects may include high blood sugar, rash, diarrhea, and nausea. Serious side effects can include severe skin reactions, inflammation of the lungs, and pancreatitis (inflammation of the pancreas). Blood sugar levels will be closely monitored, and management strategies will be in place for those who develop high blood sugar. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT04524000?
Ask your doctor about the specific genetic tests needed to confirm the PIK3CA mutation and HER2 status. Understand the potential side effects of alpelisib and fulvestrant, and how they will be managed during the trial. Be prepared for regular clinic visits for drug administration, blood tests, and imaging scans to monitor your cancer and overall health. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT04524000 signal from an investment perspective?
This trial focuses on a specific genetic subtype of breast cancer, a growing area of targeted therapy, suggesting potential for a niche market if successful, though competition in advanced breast canc This is a Phase 2 trial, which is focused on confirming efficacy before larger pivotal studies.
What happens if the treatment in this trial doesn't work?
Participants will receive the study drugs, and their health will be closely monitored through regular visits and tests. 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.
Related Conditions
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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.