Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Have Received Alpelisib as Part of a Compassionate Use Program (EPIK-P1)
ClinicalTrials.gov Identifier: NCT04285723
Novartis Reference Number: CBYL719F12002
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.
Study Description
The study is a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS). Patient-level data are abstracted from medical charts of all eligible patients at all participating sites.
Information from patients treated with alpelisib will be used to describe the efficacy and safety of alpelisib in PROS patients.
Eligibility Criteria
Inclusion Criteria:
Patient (adult or pediatric) is ≥ 2 years of age *
Patient has a physician confirmed/documented diagnosis of PROS*
Patient has a documented evidence of a mutation in the PIK3CA gene*
Patient's condition was assessed by the treating physician as severe or life threatening and treatment was deemed necessary*
Patient has been treated with at least one dose of alpelisib, initiated on or before 23-Sep-2019 (i.e. at least 24 weeks before the cut-off date of the 09-Mar-2020)
Patient has medical chart history available during enrollment in the Novartis MAP
Patient (or parent/guardian in case of pediatric patient) consented to participate in the study (as required by local ethics regulations) * Inclusion criteria for MAP enrollment (assessed at the time of alpelisib initiation)
Study Locations
Contacts
Have a question?
Call 1-999-669-6682 or email [email protected]