drugset / Trial / NCT06997588
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)
NaParallel-groupOpen-labelTreatment
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).
Timeline
- Start
- 2025-10-09
- Primary completion
- 2028-08-02
- Completion
- 2030-09-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Alpelisib | Small molecule | 50 mg | Oral |
| Subject | Alpelisib | Small molecule | 125 mg | Oral |
| Subject | Alpelisib | Small molecule | 250 mg | Oral |