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
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 4
- 主要终点
- Incidence proportion of hyperglycemia
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Incidence proportion of hyperglycemia
时间窗: 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.
次要结局
- Calculated BMI(Baseline)
- Number of participants with incidence proportion of ketoacidosis and Hyperglycemic Hyperosmolar Non-Ketotic Syndrome (HHNKS)(Up to 53 months)
- Incidence of the Adverse Events of Special Interest (AESI) of Osteonecrosis of the Jaw (ONJ)(Up to 53 months)
- Medical history(Baseline)
- Family history of diabetes mellitus(Baseline)
- Number of participants with risk factors for Osteonecrosis of the Jaw (ONJ) observed(Baseline)
- Incidence proportion of AESIs(Up to 53 months)
- Other safety and tolerability events(Up to 53 months)
- Number of patients with hematological and biochemical laboratory abnormalities(Up to 53 months)
- Number of patients with concomitant medications known to affect blood glucose levels(Baseline)