Clinical Trial

EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)

Recruiting Phase 2
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Summary
This study is designed to demonstrate the efficacy and assess safety and tolerability of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).
Protocol Amendment History 1 change
notable Trial sites expanded: 22 -> 27 locations 2026-04-02
Trial Details
NCT Number NCT06997588
Lead Sponsor Novartis Pharmaceuticals
Conditions PIK3CA-related Overgrowth Spectrum (PROS)
Enrollment 104 participants
Start Date 2025-10-09
Primary Completion 2028-08-02 (estimated)
Study Completion 2030-09-30 (estimated)
Updated on ClinicalTrials.gov 2026-07-08