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, in the real-world setting

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  PIQRAY
• Study drug International non-proprietary name (INN) or common name:
  ALPELISIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01EM03) alpelisib

  alpelisib
• Medical condition to be studied:
  Breast cancer
• Additional medical condition(s):
  Hormone receptor positive HER2 negative breast cancer

Population studied

• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  150

Study design details

• Main study objective:
  The main objectives of this study is to evaluate the safety of alpelisib in combination with fulvestrant in the described population in the real-world setting. ; As per the risk management plan approved by the European Medicines Agency, this study will primarily focus on the risk of hyperglycemia and the risk factors for hyperglycemia.
• Outcomes:
  - To Assess the incidence of hyperglycemia (adverse event of special interest, AESI) observed during follow-up of patients treated with alpelisib in combination with fulvestrant, ; - To assess the risk factors of hyperglycemia, ; - To estimate the incidence of complications of a non-compensated hyperglycemic state, such as ketoacidosis and hyperglycemic hyperosmolar non-ketotic syndrome, ; - To assess the incidence of osteonecrosis of the jaw, and the risk factors for ONJ, To describe other AESIs, including GI toxicity, rash, hypersensitivity, pancreatitis, pneumonitis and SCARs
• Data analysis plan:
  Data analysis will include: ; • cumulative incidence proportions estimated at different time points to assess specific events of interest ; • descriptive analyses of relevant risk factors ; • logistic regression to assess risk factors where appropriate ; • where relevant, risk factors will be assessed taking time into consideration ; • overall number and incidence proportion of patients with adverse events of special interest, will be summarized with 95% confidence intervals ; • no statistical hypotheses will be tested in this study ; • where inferential statistical methods are used their results are considered to be purely descriptive.