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

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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.

Condition 
PIK3CA-Related Overgrowth Spectrum (PROS)
Phase 
Not Given
Overall status 
Recruiting
Enrollment count 
65 participants
Start date 
Jun 09, 2020
Completion date 
Sep 29, 2020
Gender 
All
Age(s)
2 Years and older (Child, Adult, Older Adult)

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

United States
Boston Childrens Hospital
Recruiting
Boston, 02215
Massachusetts
United States
Australia
Novartis Investigative Site
Recruiting
Parkville, 3052
Victoria
Australia
France
Novartis Investigative Site
Recruiting
Montpellier cedex 5, 34059
Herault
France
Novartis Investigative Site
Recruiting
Dijon, 21000
-
France
Novartis Investigative Site
Recruiting
Paris cedex 15, 75015
-
France
Ireland
Novartis Investigative Site
Recruiting
Dublin, 12
-
Ireland
Spain
Novartis Investigative Site
Recruiting
Madrid, 28046
-
Spain

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
1-888-669-6682
Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Email: 

Have a question?

Call 1-999-669-6682 or email [email protected]