Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
Prospective period only: Proportion of participants with new or worsening grade =3 treatment emergent adverse events (AEs) |
Incidence of new or worsening grade =3 treatment emergent AEs (by system organ class and preferred term) |
From date of first interventional dose administration in the prospective period (Day 1) to 30 days after last dose of study drug, assessed up to 5 years. |
|
Secondary |
Retrospective period: Proportion of participants with Adverse Events (AEs) |
Incidence, type and severity per common terminology criteria for AEs (CTCAE) v4.03 criteria, causality assessments of AEs, including changes in laboratory values, vital signs and assessment of cardiac function during the retrospective period. |
From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1) |
|
Secondary |
Prospective period: Proportion of participants with AEs |
Incidence, type and severity per CTCAE v4.03 criteria, causality assessments of AEs, including changes in laboratory values, vital signs, assessment of cardiac function, and growth, sexual maturation and bone/dental development (for applicable age) during the prospective period. |
From Day 1 up to 5 years |
|
Secondary |
Retrospective and prospective period: Overall clinical assessment as assessed by the investigator |
Proportion of participants with overall clinical assessment reported as improvement, stable or worsened, as assessed by the investigator. |
Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years |
|
Secondary |
Retrospective and prospective period: Incidence of PROS-related symptoms and complications/comorbidities among participants with symptoms and complications/comorbidities. |
Incidence of symptoms and complications/comorbidities (including taken treatment measures) associated with PROS over time among participants with symptoms and complications/comorbidities. |
Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years |
|
Secondary |
Retrospective and prospective period: Proportion of participants with healthcare visits/hospitalizations due to PROS. |
Proportion of participants with healthcare visits/hospitalizations due to PROS will be assessed. |
Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years |
|
Secondary |
Retrospective and prospective period: Proportion of participants requiring PROS-related treatment(s) other than alpelisib |
Proportion of participants requiring PROS-related treatment(s) other than alpelisib, including medications (concomitant PROS-related medications including medication for the management of PROS related complications as well as medications to manage complications secondary to alpelisib) and non-drug treatments (e.g., feeding tube, ketogenic diet, non-invasive device for sleep apnea, sclerotherapy, endovascular occlusive procedures) |
Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years |
|
Secondary |
Retrospective and prospective period: Proportion of participants with dose adjustments of alpelisib |
Proportion of participants requiring dose increase, reductions and interruptions of alpelisib |
Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years |
|
Secondary |
Retrospective and prospective period: Proportion of participants with PROS-related surgeries |
Proportion of participants requiring surgery due to PROS |
Retrospective: From 10-Mar-2020 (1 day after EPIK-P1 data cut-off date) to the day before the first interventional dose of alpelisib is administered in the prospective period of this study (Day -1). Prospective: From Day 1 up to 5 years |
|