Clinical trial NCT04967248: 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
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
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Hormone Receptor Positive HER2 Negative Breast Cancer With a PIK3CA Mutation
Keywords: Breast Cancer
non-interventional study
NIS
alpelisib
fulvestrant
PASS
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2021-07-19
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Postmenopausal women, and adult men, with HR+, HER2- locally advanced or metastatic breast cancer whose tumor harbors a PIK3CA mutation, and who are treated with alpelisib in combination with fulvestrant after disease progression following endocrine therapy as monotherapy, in the real-world setting.

Brief summary: This is 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 are 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 provides informed consent, he or she is enrolled in the study. Patients will 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 occurs first. The end of the study is defined as a maximum of 12 months after the date the last patient was enrolled (LPFV); if the last patient is still on treatment on that date, they will not be followed up any further

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Incidence proportion of hyperglycemia
timeFrame: Up to 53 months
Secondary outcomes:
measure: Calculated BMI
timeFrame: Baseline
measure: Medical history
timeFrame: Baseline
measure: Family history of diabetes mellitus
timeFrame: Baseline
measure: Number of patients with concomitant medications known to affect blood glucose levels
timeFrame: Baseline
measure: Number of participants with incidence proportion of ketoacidosis and Hyperglycemic Hyperosmolar Non-Ketotic Syndrome (HHNKS)
timeFrame: Up to 53 months
measure: Incidence of the Adverse Events of Special Interest (AESI) of Osteonecrosis of the Jaw (ONJ)
timeFrame: Up to 53 months
measure: Number of participants with risk factors for Osteonecrosis of the Jaw (ONJ) observed
timeFrame: Baseline
measure: Incidence proportion of AESIs
timeFrame: Up to 53 months
measure: Other safety and tolerability events
timeFrame: Up to 53 months
measure: Number of patients with hematological and biochemical laboratory abnormalities
timeFrame: Up to 53 months
Other outcomes:
Locations:
city: Pavia
country: Italy
facility: Novartis Investigative Site
state: PV
city: Bari
country: Italy
facility: Novartis Investigative Site
city: Potenza
country: Italy
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: