Hormone Receptor Positive HER2 Negative Breast Cancer With a PIK3CA MutationNCT04967248
A NIS of Alpelisib in Combination With Fulvestrant in Postmenopausal Women, and Men, With HR+,HER2- , Locally Advanced or Metastatic Breast Cancer With a PIK3CA Mutation, After Disease Progression Following Endocrine Therapy as Monotherapy, in the Real-world Setting
An observational study of Hormone Receptor Positive HER2 Negative Breast Cancer With a PIK3CA Mutation, run by Novartis Pharmaceuticals.
Trial summary
This was a prospective, multi-national, non-interventional study (NIS) collecting data from postmenopausal women, and adult men, with HR+, HER2- locally advanced or metastatic breast cancer whose tumor harbors a PIK3CA mutation, and who were to be treated with alpelisib in combination with fulvestrant after disease… Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Hormone Receptor Positive HER2 Negative Breast Cancer With a PIK3CA Mutation
- What is being tested?
- Alpelisib and Fulvestrant
- How many people
- 4 participants
- Where
- No sites listed yet
- Study dates
- 21 Jun 2023to 18 Oct 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Postmenopausal women, and adult men, with HR+, HER2- locally advanced or metastatic breast cancer whose tumor harbors a PIK3CA mutation, and who were treated with alpelisib in combination with fulvestrant after disease progression following endocrine therapy as monotherapy, in the real-world setting.
Who may be able to join
- Signed informed consent from the patient or a legally acceptable representative, obtained before any study-related activities are undertaken
- Patients diagnosed with HR+, HER2- locally advanced or metastatic breast cancer with a PIK3CA mutation
- Patients who have disease progression following endocrine therapy as monotherapy
- Patients must be postmenopausal women, or men, ≥18 years of age
- Patients recruited on or before their first prescribed dose of alpelisib in combination with fulvestrant
Who cannot join
- Use of alpelisib prior to signing the informed consent form for this study
- Participation in an interventional study within 30 days prior to the initiation of alpelisib
About this study
Official title: Alpelisib (Piqray®) Post-Authorization Safety Study (PASS): a Non-interventional Study of Alpelisib in Combination With Fulvestrant in Postmenopausal Women, and Men, With Hormone Receptor Positive (HR+), Human Epidermal Growth Factor Receptor 2 Negative (HER2-), Locally Advanced or Metastatic Breast Cancer With a Phosphatidylinositol-3-kinase Catalytic Subunit Alpha (PIK3CA) Mutation, After Disease Progression Following Endocrine Therapy as Monotherapy, in the Real-world Setting
This was a prospective, multi-national, non-interventional study (NIS) collecting data from postmenopausal women, and adult men, with HR+, HER2- locally advanced or metastatic breast cancer whose tumor harbors a PIK3CA mutation, and who were to be treated with alpelisib in combination with fulvestrant after disease progression following endocrine therapy as monotherapy, in the real-world setting.
Read the detailed description
Once the patient provided informed consent, he or she was enrolled in the study. Patients were planned to be followed from enrollment until 1) 30 days after alpelisib treatment discontinuation, or 2) death, or 3) lost to follow-up, or 4) patient withdrawal, or 5) physician decision to end treatment/study, or 6) end of the study, whichever occured first. The end of the study was defined as a maximum of 12 months after the date the last patient was enrolled (LPFV); if the last patient was still on treatment on that date, they were not be followed up any further.
Due to very low patient numbers (4 patients, including 2 eligible patients) no statistical analyses were performed. Database lock was achieved without all queries resolved.
- Being tested
- Alpelisib, Fulvestrant
- Keywords
- Breast Cancer, non-interventional study, NIS, alpelisib, fulvestrant, PASS
- Medical subject headings
- Breast Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlpelisibOther
Prospective observational PASS study. There was no treatment allocation. Patients recruited on or before their first prescribed dose of alpelisib in combination with fulvestrant were enrolled.
FulvestrantOther
Prospective observational PASS study. There was no treatment allocation. Patients recruited on or before their first prescribed dose of alpelisib in combination with fulvestrant were enrolled.
| Study group | Role | What they receive |
|---|---|---|
| alpelisib in combination with fulvestrant | Patients treated with alpelisib in combination with fulvestrant Receives: Other: Alpelisib; Other: Fulvestrant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence proportion of hyperglycemia
To assess the incidence of hyperglycemia (Adverse Event of Special Iinterest) observed during follow-up of patients treated with alpelisib in combination with fulvestrant.
Measured: Up to 53 months
Secondary outcomes (10)
Calculated BMI
Calculated BMI will be collected
Measured: Baseline
Medical history
Number of patients with diabetes mellitus (including gestational diabetes), tobacco use, baseline diabetic status per laboratory values for HbA1c and FPG
Measured: Baseline
Family history of diabetes mellitus
Yes/ No variable
Measured: Baseline
Number of patients with concomitant medications known to affect blood glucose levels
Number of patients with concomitant medications known to affect blood glucose levels will be measured
Measured: Baseline
Number of participants with incidence proportion of ketoacidosis and Hyperglycemic Hyperosmolar Non-Ketotic Syndrome (HHNKS)
Number of participants with incidence proportion of ketoacidosis and HHNKS based on AE data
Measured: Up to 53 months
Incidence of the Adverse Events of Special Interest (AESI) of Osteonecrosis of the Jaw (ONJ)
Incidence of AESI of Osteonecrosis of the Jaw (ONJ) will be provided
Measured: Up to 53 months
Number of participants with risk factors for Osteonecrosis of the Jaw (ONJ) observed
Number of participants with risk factors for ONJ will be collected. Risk factors for ONJ include:
- Patient characteristics: age, calculated BMI, sex
- Prior and/or concomitant use of bisphosphonates (e.g. zoledronic acid).
- Prior and/or concomitant use of RANKligand inhibitors (e.g. denosumab).
Measured: Baseline
Incidence proportion of AESIs
The incidence proportion of AESIs:
- GI toxicity (nausea, vomiting and diarrhea)
- Rash
- Hypersensitivity (e.g. anaphylactic reaction)
- Pancreatitis
- Pneumonitis
- SCARs
Measured: Up to 53 months
Other safety and tolerability events
The incidence proportion of:
- AEs
- AEs leading to dose interruptions
- AEs leading to dose reductions
- AEs leading to permanent discontinuation of alpelisib in combination with fulvestrant
- SAEs
Measured: Up to 53 months
Number of patients with hematological and biochemical laboratory abnormalities
Number of patients with hematological and biochemical laboratory abnormalities will be provided
Measured: Up to 53 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2021
- Study start21 Jun 2023
- Primary completion18 Oct 2024
- Study completion18 Oct 2024
Why it stopped: Low recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hormone Receptor Positive HER2 Negative Breast Cancer With a PIK3CA MutationStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04967248
- Sponsor's protocol ID
- CBYL719C2404
- Status last verified
- Oct 2025
- First submitted
- 8 Jul 2021
- Last updated
- 4 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Alpelisib (Piqray®) Post-Authorization Safety Study (PASS): a Non-interventional Study of Alpelisib in Combination With Fulvestrant in Postmenopausal Women, and Men, With Hormone Receptor Positive (HR+), Human Epidermal Growth Factor Receptor 2 Negative (HER2-), Locally Advanced or Metastatic Breast Cancer With a Phosphatidylinositol-3-kinase Catalytic Subunit Alpha (PIK3CA) Mutation, After Disease Progression Following Endocrine Therapy as Monotherapy, in the Real-world Setting
- ClinicalTrials.gov ID
- NCT04967248
- Acronym / sponsor ID
- CBYL719C2404
- Keywords
- Breast Cancer, non-interventional study, NIS, alpelisib, fulvestrant, PASS
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Low recruitment
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 21 Jun 2023 (actual)
- Primary completion
- 18 Oct 2024 (actual)
- Study completion
- 18 Oct 2024 (actual)
- First posted
- 19 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a prospective, multi-national, non-interventional study (NIS) collecting data from postmenopausal women, and adult men, with HR+, HER2- locally advanced or metastatic breast cancer whose tumor harbors a PIK3CA mutation, and who were to be treated with alpelisib in combination with fulvestrant after disease progression following endocrine therapy as monotherapy, in the real-world setting.
Detailed description
Once the patient provided informed consent, he or she was enrolled in the study. Patients were planned to be followed from enrollment until 1) 30 days after alpelisib treatment discontinuation, or 2) death, or 3) lost to follow-up, or 4) patient withdrawal, or 5) physician decision to end treatment/study, or 6) end of the study, whichever occured first. The end of the study was defined as a maximum of 12 months after the date the last patient was enrolled (LPFV); if the last patient was still on treatment on that date, they were not be followed up any further.
Due to very low patient numbers (4 patients, including 2 eligible patients) no statistical analyses were performed. Database lock was achieved without all queries resolved.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| alpelisib in combination with fulvestrant | – | Other: Alpelisib, Other: FulvestrantPatients treated with alpelisib in combination with fulvestrant |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Postmenopausal women, and adult men, with HR+, HER2- locally advanced or metastatic breast cancer whose tumor harbors a PIK3CA mutation, and who were treated with alpelisib in combination with fulvestrant after disease progression following endocrine therapy as monotherapy, in the real-world setting.
Key inclusion criteria (5 listed)
- Signed informed consent from the patient or a legally acceptable representative, obtained before any study-related activities are undertaken
- Patients diagnosed with HR+, HER2- locally advanced or metastatic breast cancer with a PIK3CA mutation
- Patients who have disease progression following endocrine therapy as monotherapy
- Patients must be postmenopausal women, or men, ≥18 years of age
- Patients recruited on or before their first prescribed dose of alpelisib in combination with fulvestrant
Key exclusion criteria (2 listed)
- Use of alpelisib prior to signing the informed consent form for this study
- Participation in an interventional study within 30 days prior to the initiation of alpelisib
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AlpelisibProspective observational PASS study. There was no treatment allocation. Patients recruited on or before their first prescribed dose of alpelisib in combination with fulvestrant were enrolled. | Other | alpelisib in combination with fulvestrant |
| FulvestrantProspective observational PASS study. There was no treatment allocation. Patients recruited on or before their first prescribed dose of alpelisib in combination with fulvestrant were enrolled. | Other | alpelisib in combination with fulvestrant |
Primary outcome
Incidence proportion of hyperglycemia
Time frame: Up to 53 months
To assess the incidence of hyperglycemia (Adverse Event of Special Iinterest) observed during follow-up of patients treated with alpelisib in combination with fulvestrant.
10 secondary outcomes
Calculated BMI
Time frame: Baseline
Medical history
Time frame: Baseline
Family history of diabetes mellitus
Time frame: Baseline
Number of patients with concomitant medications known to affect blood glucose levels
Time frame: Baseline
Number of participants with incidence proportion of ketoacidosis and Hyperglycemic Hyperosmolar Non-Ketotic Syndrome (HHNKS)
Time frame: Up to 53 months
Incidence of the Adverse Events of Special Interest (AESI) of Osteonecrosis of the Jaw (ONJ)
Time frame: Up to 53 months
Number of participants with risk factors for Osteonecrosis of the Jaw (ONJ) observed
Time frame: Baseline
Incidence proportion of AESIs
Time frame: Up to 53 months
Other safety and tolerability events
Time frame: Up to 53 months
Number of patients with hematological and biochemical laboratory abnormalities
Time frame: Up to 53 months
Results and safety
The study was stopped early (reason given: Low recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2025.
- Link to study resultsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
An observational study of Hormone Receptor Positive HER2 Negative Breast Cancer With a PIK3CA Mutation, run by Novartis Pharmaceuticals.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 4 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Low recruitment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence proportion of hyperglycemia (time frame: Up to 53 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2025.