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PI3K Inhibitor

Alpelisib + Endocrine Therapy for Breast Cancer

Phase 2
Recruiting
Led By Amanda Parkes, MD
Research Sponsored by Amanda Parkes
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age ≥ 18 years at the time of consent.
PIK3CA mutation identified via local testing from tumor tissue or blood.
Must not have
Active cardiac disease, defined as any of the following within 6 months prior to the start of study treatment: History of angina pectoris, coronary artery bypass graft, symptomatic pericarditis, or myocardial infarction. History of documented congestive heart failure (New York Heart Association functional classification III-IV). History of any cardiac arrhythmias, (e.g., ventricular tachycardia), complete left bundle branch block, high grade AV block (e.g., bifascicular block, Mobitz type II and third degree AV block), supraventricular, nodal arrhythmias, or conduction abnormality. Systolic blood pressure (SBP) >180 mmHg and/or Diastolic Blood Pressure (DBP) ≥ 100 mm Hg at screening. Initiation or adjustment of antihypertensive medication(s) is allowed prior to screening.
Prior treatment with PI3K, mTOR or AKT inhibitor in the metastatic setting.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from enrollment until the time of death, up to 60 months
Awards & highlights
No Placebo-Only Group

Summary

This trial is for patients with late-stage breast cancer who have a specific genetic mutation. They will take a daily drug (alpelisib) in combination with a standard cancer treatment. The trial will follow patients to see how well they respond to the new drug.

Who is the study for?
This trial is for adults with hormone receptor positive, HER2 negative, PIK3CA mutant metastatic breast cancer that has progressed after endocrine therapy. Participants must have an ECOG Performance Status of 0-2 and adequate organ function. Men and premenopausal women must agree to contraception. Exclusions include prior chemotherapy for advanced disease, other malignancies, CNS metastases causing symptoms, uncontrolled diseases or infections.
What is being tested?
The study tests alpelisib in combination with ongoing endocrine therapy (Fulvestrant or Aromatase Inhibitor) in patients whose breast cancer has worsened on just endocrine therapy. It's a multi-phase trial including screening, treatment until disease progression/toxicity, and post-treatment follow-up to assess safety and effectiveness.
What are the potential side effects?
Alpelisib can cause high blood sugar levels which may need medical attention. Other side effects might include rash, nausea, fatigue and diarrhea. Fulvestrant may cause injection site reactions while aromatase inhibitors can lead to bone loss or joint pain.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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My cancer has a PIK3CA mutation.
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My breast cancer is confirmed to be HER2 negative.
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My breast cancer cannot be removed by surgery and has spread.
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I have been treated with hormone therapy for my cancer that has spread.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had serious heart issues or very high blood pressure in the last 6 months.
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I have been treated with PI3K, mTOR, or AKT inhibitors for cancer that has spread.
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I have received chemotherapy for my advanced or metastatic disease.
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I do not have brain metastases causing symptoms or needing treatment.
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I have been on daily steroids for more than 14 days and need to continue.
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I have had acute pancreatitis in the last year or suffer from chronic pancreatitis.
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I have type 1 diabetes or poorly controlled diabetes with A1c > 8.0% for over a year.
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I do not have another cancer that could affect this trial's safety or results.
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I am currently on medication for an infection.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from enrollment until the time of death, up to 60 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and from enrollment until the time of death, up to 60 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Progression-Free Survival (PFS)
Secondary study objectives
Clinical Benefit Rate (CBR)
Duration of Response (DOR)
Objective Response Rate (ORR)
+2 more

Side effects data

From 2023 Phase 3 trial • 572 Patients • NCT02437318
62%
Hyperglycaemia
57%
Diarrhoea
45%
Nausea
35%
Rash
35%
Decreased appetite
26%
Vomiting
26%
Weight decreased
24%
Fatigue
24%
Stomatitis
20%
Asthenia
20%
Alopecia
18%
Mucosal inflammation
18%
Pruritus
17%
Dysgeusia
17%
Headache
15%
Dry skin
14%
Oedema peripheral
14%
Pyrexia
14%
Back pain
13%
Rash maculo-papular
11%
Dyspepsia
11%
Abdominal pain
11%
Arthralgia
10%
Dry mouth
10%
Urinary tract infection
10%
Blood creatinine increased
10%
Gamma-glutamyltransferase increased
10%
Aspartate aminotransferase increased
10%
Cough
8%
Nasopharyngitis
8%
Pain in extremity
8%
Dizziness
8%
Hypertension
8%
Anaemia
8%
Constipation
8%
Alanine aminotransferase increased
8%
Hypokalaemia
8%
Dyspnoea
7%
Myalgia
7%
Muscle spasms
7%
Insomnia
6%
Lipase increased
6%
Abdominal pain upper
5%
Lymphoedema
5%
Musculoskeletal pain
5%
Erythema
4%
Upper respiratory tract infection
4%
Bone pain
3%
Hot flush
2%
Osteonecrosis of jaw
2%
Acute kidney injury
1%
Pneumonitis
1%
Dehydration
1%
Pulmonary embolism
1%
Upper gastrointestinal haemorrhage
1%
General physical health deterioration
1%
Cellulitis
1%
Pleural effusion
1%
Hypersensitivity
1%
Pneumonia
1%
Hyponatraemia
1%
Muscular weakness
1%
Brain oedema
1%
Renal failure
1%
Erythema multiforme
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo qd + Fulvestrant
Alpelisib qd + Fulvestrant

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Alpelisib + Aromatase Inhibitor or FulvestrantExperimental Treatment3 Interventions
Subjects will be treated with Alpelisib in combination with either an Aromatase Inhibitor or Fulvestrant per Standard of Care
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Alpelisib
2018
Completed Phase 3
~960
Fulvestrant
2011
Completed Phase 3
~3790
Aromatase inhibitor
2021
Completed Phase 4
~5510

Find a Location

Who is running the clinical trial?

Amanda ParkesLead Sponsor
Marina N SharifiLead Sponsor
NovartisIndustry Sponsor
1,639 Previous Clinical Trials
2,774,259 Total Patients Enrolled
58 Trials studying Breast Cancer
21,263 Patients Enrolled for Breast Cancer
University of Wisconsin, MadisonOTHER
1,237 Previous Clinical Trials
3,200,935 Total Patients Enrolled
36 Trials studying Breast Cancer
38,481 Patients Enrolled for Breast Cancer
Kari WisinskiLead Sponsor
2 Previous Clinical Trials
74 Total Patients Enrolled
Amanda Parkes, MDPrincipal InvestigatorUniversity of Wisconsin, Madison
Kari Wisinski, MDPrincipal InvestigatorUniversity of Wisconsin, Madison
6 Previous Clinical Trials
125 Total Patients Enrolled
2 Trials studying Breast Cancer
35 Patients Enrolled for Breast Cancer
Marina N Sharifi, MDPrincipal InvestigatorUniversity of Wisconsin, Madison

Media Library

Alpelisib (PI3K Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04762979 — Phase 2
Breast Cancer Research Study Groups: Alpelisib + Aromatase Inhibitor or Fulvestrant
Breast Cancer Clinical Trial 2023: Alpelisib Highlights & Side Effects. Trial Name: NCT04762979 — Phase 2
Alpelisib (PI3K Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04762979 — Phase 2
~7 spots leftby Sep 2025