Hormone Receptor Positive HER2 Negative Breast Cancer With a PIK3CA Mutation
Conditions
Keywords
Breast Cancer, non-interventional study, NIS, alpelisib, fulvestrant, PASS
Brief 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 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.
Interventions
OTHERAlpelisib
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.
OTHERFulvestrant
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.
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 100 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence proportion of hyperglycemia | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Medical history | Baseline | Number of patients with diabetes mellitus (including gestational diabetes), tobacco use, baseline diabetic status per laboratory values for HbA1c and FPG |
| Family history of diabetes mellitus | Baseline | Yes/ No variable |
| Number of patients with concomitant medications known to affect blood glucose levels | Baseline | Number of patients with concomitant medications known to affect blood glucose levels will be measured |
| Number of participants with incidence proportion of ketoacidosis and Hyperglycemic Hyperosmolar Non-Ketotic Syndrome (HHNKS) | Up to 53 months | Number of participants with incidence proportion of ketoacidosis and HHNKS based on AE data |
| Calculated BMI | Baseline | Calculated BMI will be collected |
| Number of participants with risk factors for Osteonecrosis of the Jaw (ONJ) observed | Baseline | 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). |
| Incidence proportion of AESIs | Up to 53 months | The incidence proportion of AESIs: * GI toxicity (nausea, vomiting and diarrhea) * Rash * Hypersensitivity (e.g. anaphylactic reaction) * Pancreatitis * Pneumonitis * SCARs |
| Other safety and tolerability events | Up to 53 months | 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 |
| Number of patients with hematological and biochemical laboratory abnormalities | Up to 53 months | Number of patients with hematological and biochemical laboratory abnormalities will be provided |
| Incidence of the Adverse Events of Special Interest (AESI) of Osteonecrosis of the Jaw (ONJ) | Up to 53 months | Incidence of AESI of Osteonecrosis of the Jaw (ONJ) will be provided |
Outcome results
None listed