EPIK-P3: A phase II study to evaluate the long-term safety and efficacy of alpelisib in patients with PIK3CA Related-Overgrowth Spectrum (PROS) who previously participated in Study CBYL719F12002 (EPIK-P1)

Conditions

PIK3CA-Related Overgrowth Spectrum (PROS)

Brief summary

Incidence of new or worsening grade ≥3 treatment emergent AEs (by system organ class and preferred term)

Detailed description

Retrospective Period Only - Incidence, type and severity per common terminology criteria for AEs (CTCAE) v4.03 criteria, causality assessments of AEs, and other safety data including changes in laboratory values, vital signs and assessment of cardiac function., Prospective Period Only - Incidence, type and severity per CTCAE v4.03 criteria, causality assessments of AEs, and other safety data including changes in laboratory values, vital signs and assessment of cardiac function. Additionally, for this period only, growth, bone/dental development and sexual maturation (for applicable age) will be assessed., Investigator assessment of lesion response as improved, stable or worsened., Incidence of PROS-related symptoms and complications/comorbidities among participants with symptoms and complications/comorbidities., Number of healthcare visits/hospitalizations due to PROS., Description of medications and non-drug therapies received -PROS-related treatment(s) other than alpelisib -Medication(s) (e.g., concomitant PROS-related medications including medication for the management of PROS related complications as well as medications to manage complications secondary to alpelisib) -Non-drug treatment(s) -Alpelisib treatment -PROS-related surgeries

Related papers

No related papers found yet.

Interventions

DRUGBYL719

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence of new or worsening grade ≥3 treatment emergent AEs (by system organ class and preferred term)	—

Secondary

Measure	Time frame
Retrospective Period Only - Incidence, type and severity per common terminology criteria for AEs (CTCAE) v4.03 criteria, causality assessments of AEs, and other safety data including changes in laboratory values, vital signs and assessment of cardiac function., Prospective Period Only - Incidence, type and severity per CTCAE v4.03 criteria, causality assessments of AEs, and other safety data including changes in laboratory values, vital signs and assessment of cardiac function. Additionally, for this period only, growth, bone/dental development and sexual maturation (for applicable age) will be assessed., Investigator assessment of lesion response as improved, stable or worsened., Incidence of PROS-related symptoms and complications/comorbidities among participants with symptoms and complications/comorbidities., Number of healthcare visits/hospitalizations due to PROS., Description of medications and non-drug therapies received -PROS-related treatment(s) other than alpelisib -Medicat	—

Measure

Time frame

Retrospective Period Only - Incidence, type and severity per common terminology criteria for AEs (CTCAE) v4.03 criteria, causality assessments of AEs, and other safety data including changes in laboratory values, vital signs and assessment of cardiac function., Prospective Period Only - Incidence, type and severity per CTCAE v4.03 criteria, causality assessments of AEs, and other safety data including changes in laboratory values, vital signs and assessment of cardiac function. Additionally, for this period only, growth, bone/dental development and sexual maturation (for applicable age) will be assessed., Investigator assessment of lesion response as improved, stable or worsened., Incidence of PROS-related symptoms and complications/comorbidities among participants with symptoms and complications/comorbidities., Number of healthcare visits/hospitalizations due to PROS., Description of medications and non-drug therapies received -PROS-related treatment(s) other than alpelisib -Medicat

—

Countries

France, Ireland, Spain

Outcome results

None listed