NCT04285723

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)

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 57 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

The study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS) who initiated alpelisib at least 24 weeks before the cut-off date at a MAP site. The study cut-off date was 09-Mar-2020. Patient-level data were abstracted from medical charts of all eligible patients at all participating sites. Study completion date refers to the last date data was extracted. Information from patients treated with alpelisib was used to describe the efficacy and safety of alpelisib in PROS patients.

Detailed description

The index date (baseline) is defined as the date of alpelisib initiation. The study period is the period from the index date up to the most recent data available at the time of the cut-off date. The maximum follow-up was 187 weeks.

Conditions

• PIK3CA-Related Overgrowth Spectrum (PROS)

Interventions

Type	Name	Description
OTHER	alpelisib	Retrospective observational case-only study. There is no treatment allocation. Patients with severe or life-threatening PROS who have received alpelisib as part of a compassionate use program were invited to participate.

Timeline

• Start date: 2020-06-09
• Primary completion: 2021-04-16
• Completion: 2021-04-16
• First posted: 2020-02-26
• Last updated: 2023-02-09
• Results posted: 2023-02-09

Locations

7 sites across 5 countries: United States, Australia, France, Ireland, Spain