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New hope for rare overgrowth disorder: drug trial targets root cause

NCT ID NCT06997588

First seen Nov 01, 2025 · Last updated May 24, 2026 · Updated 34 times

Summary

This study tests a daily oral drug called alpelisib in children and adults with PROS, a rare condition causing abnormal tissue growth. The goal is to see if the drug can shrink overgrowths and improve symptoms. Participants must be at least 2 years old and have active, worsening overgrowth.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Cinn Children Hosp Medical Center

    RECRUITING

    Cincinnati, Ohio, 45206, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Novartis Investigative Site

    RECRUITING

    Sydney, New South Wales, 2010, Australia

  • Novartis Investigative Site

    RECRUITING

    Sydney, New South Wales, 2031, Australia

  • Novartis Investigative Site

    RECRUITING

    North Adelaide, South Australia, 5066, Australia

  • Novartis Investigative Site

    RECRUITING

    Salzburg, 5020, Austria

  • Novartis Investigative Site

    RECRUITING

    Vienna, A 1090, Austria

  • Novartis Investigative Site

    RECRUITING

    Ghent, 9000, Belgium

  • Novartis Investigative Site

    RECRUITING

    Angers, 49933, France

  • Novartis Investigative Site

    RECRUITING

    Bron, 69677, France

  • Novartis Investigative Site

    RECRUITING

    Dijon, 21000, France

  • Novartis Investigative Site

    RECRUITING

    Montpellier, 34295, France

  • Novartis Investigative Site

    RECRUITING

    Paris, 75015, France

  • Novartis Investigative Site

    RECRUITING

    Toulouse, 31400, France

  • Novartis Investigative Site

    RECRUITING

    Tours, 37044, France

  • Novartis Investigative Site

    RECRUITING

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Novartis Investigative Site

    RECRUITING

    Düsseldorf, North Rhine-Westphalia, 40225, Germany

  • Novartis Investigative Site

    RECRUITING

    Leipzig, Saxony, 04103, Germany

  • Novartis Investigative Site

    RECRUITING

    Halle, Saxony-Anhalt, 06120, Germany

  • Novartis Investigative Site

    RECRUITING

    Berlin, 13353, Germany

  • Novartis Investigative Site

    RECRUITING

    Stuttgart, 70374, Germany

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00168, Italy

  • Novartis Investigative Site

    RECRUITING

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    RECRUITING

    Trieste, TS, 34137, Italy

  • Novartis Investigative Site

    RECRUITING

    Esplugues, Barcelona, 08950, Spain

  • Novartis Investigative Site

    RECRUITING

    A Coruña, 15006, Spain

  • Novartis Investigative Site

    RECRUITING

    Barcelona, 08035, Spain

  • Novartis Investigative Site

    RECRUITING

    Madrid, 28046, Spain

  • Novartis Investigative Site

    RECRUITING

    Lausanne, 1011, Switzerland

  • Novartis Investigative Site

    RECRUITING

    Liverpool, L12 2AP, United Kingdom

  • Washington University

    RECRUITING

    St Louis, Missouri, 63110, United States

    Contact

    Contact Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.