RecruitingPhase 2NCT06997588

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)

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

Sponsor

Novartis Pharmaceuticals

Enrollment

104 participants

Start Date

Oct 9, 2025

Study Type

INTERVENTIONAL

Conditions

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).

Eligibility

Min Age: 2 YearsMax Age: 100 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Alpelisib for people with pik3ca-related overgrowth spectrum (pros). The study is currently recruiting participants at 35 locations. People eligible for this study include aged 2 Years to 100 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGAlpelisib

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)

Locations(38)

Washington University

St Louis, Missouri, United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Cinn Children Hosp Medical Center

Cincinnati, Ohio, United States

Nationwide Children s Hospital

Columbus, Ohio, United States

Oregon Health Sciences University

Portland, Oregon, United States

Childrens Hosp Pittsburgh UPMC

Pittsburgh, Pennsylvania, United States

Baylor College Of Medicine

Houston, Texas, United States

UT Health Science Center

Houston, Texas, United States

Childrens Hospital and Regional Medical Center

Seattle, Washington, United States

Novartis Investigative Site

Sydney, New South Wales, Australia

Novartis Investigative Site

Sydney, New South Wales, Australia

Novartis Investigative Site

North Adelaide, South Australia, Australia

Novartis Investigative Site

Salzburg, Austria

Novartis Investigative Site

Vienna, Austria

Novartis Investigative Site

Ghent, Belgium

Novartis Investigative Site

Angers, France

Novartis Investigative Site

Bron, France

Novartis Investigative Site

Dijon, France

Novartis Investigative Site

Montpellier, France

Novartis Investigative Site

Paris, France

Novartis Investigative Site

Toulouse, France

Novartis Investigative Site

Tours, France

Novartis Investigative Site

Freiburg im Breisgau, Baden-Wurttemberg, Germany

Novartis Investigative Site

Düsseldorf, North Rhine-Westphalia, Germany

Novartis Investigative Site

Leipzig, Saxony, Germany

Novartis Investigative Site

Halle, Saxony-Anhalt, Germany

Novartis Investigative Site

Berlin, Germany

Novartis Investigative Site

Stuttgart, Germany

Novartis Investigative Site

Roma, RM, Italy

Novartis Investigative Site

Torino, TO, Italy

Novartis Investigative Site

Trieste, TS, Italy

Novartis Investigative Site

Esplugues, Barcelona, Spain

Novartis Investigative Site

A Coruña, Spain

Novartis Investigative Site

Barcelona, Spain

Novartis Investigative Site

Madrid, Spain

Novartis Investigative Site

Lausanne, Switzerland

Novartis Investigative Site

Liverpool, United Kingdom

Novartis Investigative Site

London, United Kingdom

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT06997588

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