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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)

NCT06997588 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 104

Last updated 2026-04-30

No results posted yet for this study

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).

Conditions

  • PIK3CA-related Overgrowth Spectrum (PROS)

Interventions

DRUG

Alpelisib

Oral Film-Coated Tablet (FCT): * Group 1: 250 mg once daily * Group 2, 6 to \<18 years: 125 mg once daily (starting dose) Granules: • Group 2, 2 to \<6 years: 50 mg once daily (starting dose)

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-09
Primary Completion
2028-08-02
Completion
2030-09-30
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Austria
  • Belgium
  • France
  • Germany
  • Italy
  • Spain
  • Switzerland
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06997588 on ClinicalTrials.gov