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New hope for rare overgrowth disorder: daily pill could tame symptoms

NCT ID NCT06997588

Recruiting now Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 09, 2026 · Updated 2 times

Summary

This study tests a daily oral drug called alpelisib in people aged 2 and older with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. The goal is to see if the drug can shrink overgrown tissue and improve symptoms. About 104 participants will take the drug daily, and doctors will monitor changes using MRI scans.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
alpelisib (BYL719), a daily oral drug
What this could lead to
If it works, this could provide a treatment to shrink overgrown tissue and manage symptoms in people with PROS, potentially improving daily life.
What could go wrong
This is a mid-stage trial with only 104 participants, so results may not apply to everyone. The drug may cause side effects and might not work for all types of overgrowth.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

PIK3CA-related overgrowth spectrum

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)

Contacts and locations

Locations

  • Baylor College Of Medicine

    RECRUITING

    Houston, Texas, 77030, United States

  • Childrens Hosp Pittsburgh UPMC

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

  • Cinn Children Hosp Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229-3039, United States

  • 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

  • Novartis Investigative Site

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • UNC Chapel Hill

    RECRUITING

    Chapel Hill, North Carolina, 27599, United States

  • UT Health Science Center

    RECRUITING

    Houston, Texas, 77030, United States

  • Washington University

    RECRUITING

    St Louis, Missouri, 63110, United States

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