Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum

Study Summary

This is a prospective Phase II multi-center study with an initial 16-week, randomized, double-blind, placebo-controlled period, followed by two extension periods to assess the efficacy, safety and pharmacokinetics (PK) of alpelisib in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)

Conditions

Interventions / Drugs

Alpelisib Placebo

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment206 participants

Primary Completion—

Lead SponsorNovartis Pharmaceuticals

Data Retrieved2026-04-11 02:55:49.151507+00:00

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