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PI3K Inhibitor

Alpelisib + Chemotherapy for Advanced Breast Cancer (EPIK-B3 Trial)

Phase 3
Waitlist Available
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participant has either a measurable disease per RECIST 1.1 criteria or, if no measurable disease is present, then at least one predominantly lytic bone lesion or mixed lytic-blastic bone lesion with identifiable soft tissue component (that can be evaluated by CT/MRI) must be present Part B1: Participants must have measurable disease
Participant has histologically confirmed diagnosis of advanced (loco-regionally recurrent and not amenable to curative therapy), or metastatic (stage IV) TNBC
Must not have
Participant with unresolved osteonecrosis of the jaw
Participant has a history of acute pancreatitis within 1 year prior to screening or past medical history of chronic pancreatitis
Timeline
Screening 3 weeks
Treatment Varies
Follow Up on-treatment deaths: up to approximately 28 months (part a) or approximately 17 months (part b1). post-treatment survival follow-up deaths: up to approximately 7 additional months (part a) or approximately 9 additional months (part b1).
Awards & highlights
Pivotal Trial

Summary

This trial is testing a new cancer drug to see if it's safe and effective for treating advanced triple negative breast cancer in patients with either a PIK3CA mutation or PTEN loss.

Who is the study for?
This trial is for adults with advanced triple negative breast cancer (TNBC) who have had no more than one prior therapy for metastatic disease and have a specific gene mutation (PIK3CA) or loss of the PTEN gene. They must be in good physical condition, with well-functioning organs and bone marrow.
What is being tested?
The study tests if alpelisib combined with nab-paclitaxel is safe and effective for TNBC patients with PIK3CA mutations or PTEN loss. Participants are divided into parts based on their genetic status to receive either this combination or placebo.
What are the potential side effects?
Possible side effects include reactions related to the immune system, blood sugar levels changes that could affect those with diabetes, gastrointestinal issues affecting drug absorption, severe skin reactions, lung problems like pneumonitis, and potential allergic responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a tumor that can be measured or a specific type of bone lesion.
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My breast cancer is advanced or has spread and cannot be cured with surgery or radiation.
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I have had only one treatment for my cancer since it spread.
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I am fully active or can carry out light work.
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I have had only one treatment for my cancer since it spread.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have ongoing jawbone issues not yet healed.
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I have had acute pancreatitis in the last year or have chronic pancreatitis.
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I have been diagnosed with type I diabetes or my type II diabetes is not under control.
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I am allergic to alpelisib, nab-paclitaxel, or their ingredients.
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I have been treated with drugs targeting PI3K, mTOR, or AKT.
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My CNS cancer was found recently or hasn't been treated yet.
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I have been diagnosed with pneumonitis or interstitial lung disease.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~on-treatment deaths: up to approximately 28 months (part a) or approximately 17 months (part b1). post-treatment survival follow-up deaths: up to approximately 7 additional months (part a) or approximately 9 additional months (part b1).
This trial's timeline: 3 weeks for screening, Varies for treatment, and on-treatment deaths: up to approximately 28 months (part a) or approximately 17 months (part b1). post-treatment survival follow-up deaths: up to approximately 7 additional months (part a) or approximately 9 additional months (part b1). for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Overall Response Rate (ORR) Based on Local Radiology Assessments in Participants With Measurable Disease at Baseline in Study Part B1
Progression-free Survival (PFS) Per Investigator Assessment in Study Part A
Progression-free Survival (PFS) Per Investigator Assessment in Study Part B2
Secondary study objectives
Change From Baseline in the Global Health Status/QoL Scale Score of the EORTC QLQ-C30 in Study Part B2
Clinical Benefit Rate (CBR) With Confirmed Response in Study Part A
Clinical Benefit Rate (CBR) With Confirmed Response in Study Part B1
+19 more

Side effects data

From 2025 Phase 3 trial • 137 Patients • NCT04251533
73%
Hyperglycaemia
63%
Diarrhoea
48%
Anaemia
46%
Weight decreased
38%
Nausea
25%
Stomatitis
25%
Vomiting
25%
Alopecia
25%
Decreased appetite
25%
Rash
23%
Hypokalaemia
23%
Fatigue
23%
White blood cell count decreased
21%
Neutrophil count decreased
19%
Leukopenia
19%
Neutropenia
17%
Gamma-glutamyltransferase increased
17%
Asthenia
17%
Aspartate aminotransferase increased
17%
Cough
13%
Constipation
13%
Alanine aminotransferase increased
13%
Pyrexia
12%
Pruritus
12%
Back pain
12%
Blood creatinine increased
10%
Productive cough
10%
Mucosal inflammation
10%
Headache
10%
Upper respiratory tract infection
10%
Rash maculo-papular
8%
Pleural effusion
8%
Neuropathy peripheral
8%
Urinary tract infection
8%
Arthralgia
8%
Dyspnoea
8%
Oedema peripheral
8%
Blood creatine phosphokinase increased
8%
Platelet count decreased
8%
Dry skin
8%
COVID-19
6%
Hyponatraemia
6%
Hypocalcaemia
6%
Blood alkaline phosphatase increased
6%
Taste disorder
6%
Insomnia
6%
Lethargy
6%
Peripheral sensory neuropathy
6%
Lipase increased
6%
Muscle spasms
6%
Peripheral swelling
6%
Abdominal pain
6%
Malaise
6%
Dizziness
6%
Dyspepsia
4%
Breast pain
4%
Lymphoedema
4%
Hypertension
4%
Pain in extremity
4%
Dysgeusia
4%
Lymphocyte count decreased
4%
Myalgia
4%
Pneumonia
4%
Blood lactate dehydrogenase increased
4%
Hypoalbuminaemia
2%
Blood insulin increased
2%
Polyneuropathy
2%
Cataract
2%
General physical health deterioration
2%
Lymphopenia
2%
Gastrooesophageal reflux disease
2%
Haemorrhoids
2%
Lower respiratory tract infection
2%
Dehydration
2%
Hypernatraemia
2%
Abdominal pain upper
2%
Bronchial fistula
2%
Febrile neutropenia
2%
Neck pain
2%
Hypertransaminasaemia
2%
Diabetes mellitus
2%
Hyperuricaemia
2%
Sepsis
2%
Haemorrhage intracranial
2%
Musculoskeletal chest pain
2%
Hydrothorax
2%
Appendicitis perforated
2%
Pain
2%
Device related infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part A: Alpelisib + Nab-paclitaxelEdit
Part A: Placebo + Nab-paclitaxel (Post-treatment Survival Follow-up)
Part B1: Alpelisib + Nab-paclitaxelEdit
Part A: Alpelisib + Nab-paclitaxel (Post-treatment Survival Follow-up)
Part B1: Alpelisib + Nab-paclitaxel (Post-treatment Survival Follow-up)
Part A: Placebo + Nab-paclitaxelEdit

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: Part B1: alpelisib + nab-paclitaxelExperimental Treatment2 Interventions
Participants received alpelisib 300 mg orally + nab-paclitaxel 100 mg/m\^2 IV on Days 1, 8, and 15 of each 28-day cycle.
Group II: Part A: alpelisib + nab-paclitaxelExperimental Treatment2 Interventions
Participants received alpelisib 300 mg orally + nab-paclitaxel 100 mg/m\^2 intravenously (IV) on Days 1, 8, and 15 of each 28-day cycle
Group III: Part A: placebo + nab-paclitaxelPlacebo Group2 Interventions
Participants received placebo + nab-paclitaxel 100 mg/m\^2 IV on Days 1, 8, and 15 of each 28-day cycle.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
nab-paclitaxel
2008
Completed Phase 4
~1420
alpelisib
2020
Completed Phase 3
~620

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,921 Previous Clinical Trials
4,254,175 Total Patients Enrolled
88 Trials studying Breast Cancer
37,756 Patients Enrolled for Breast Cancer

Media Library

Alpelisib (PI3K Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04251533 — Phase 3
Breast Cancer Research Study Groups: Part A: alpelisib + nab-paclitaxel, Part A: placebo + nab-paclitaxel, Part B1: alpelisib + nab-paclitaxel
Breast Cancer Clinical Trial 2023: Alpelisib Highlights & Side Effects. Trial Name: NCT04251533 — Phase 3
Alpelisib (PI3K Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04251533 — Phase 3
Breast Cancer Patient Testimony for trial: Trial Name: NCT04251533 — Phase 3
~25 spots leftby Jan 2026