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New drug shrinks Fluid-Filled growths in Gene-Linked disorder?

NCT ID NCT05948943

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

First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 25 times

Summary

This study tests whether alpelisib, a drug that targets a specific gene mutation (PIK3CA), can shrink lymphatic malformations—abnormal fluid-filled growths—and improve symptoms in children and adults. About 232 participants will receive either alpelisib or a placebo, and doctors will measure changes using MRI scans and patient-reported severity. The goal is to see if the drug can control the disease, but ongoing treatment may be needed.

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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

  • Baylor College Of Medicine

    RECRUITING

    Houston, Texas, 77030, United States

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

    Contact

  • CHOP Abramson Pediatric Resch Ctr

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact

    Contact Email: •••••@•••••

  • Childrens Hosp Boston Dept of Heme

    RECRUITING

    Boston, Massachusetts, 02115, United States

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

    Contact

  • Childrens Hosp Pittsburgh UPMC

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

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

    Contact

  • Childrens Hospital and Regional Medical Center

    RECRUITING

    Seattle, Washington, 98105, United States

    Contact Phone: •••-•••-••••

    Contact

  • Childrens National Medical Center

    RECRUITING

    Washington D.C., District of Columbia, 20010-2970, United States

    Contact Email: •••••@•••••

    Contact

  • Cinn Children Hosp Medical Center

    RECRUITING

    Cincinnati, Ohio, 45206, United States

    Contact

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

  • Cleveland Clinic Foundation

    RECRUITING

    Cleveland, Ohio, 44195, United States

    Contact Email: •••••@•••••

    Contact

  • Lucile Packard Childrens Hosp

    RECRUITING

    Palo Alto, California, 94304, United States

    Contact

    Contact Email: •••••@•••••

  • Nationwide Children s Hospital

    RECRUITING

    Columbus, Ohio, 43205, United States

    Contact

    Contact Email: •••••@•••••

  • Nemours Childrens Clinic

    RECRUITING

    Jacksonville, Florida, 32207, United States

    Contact

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

  • Novartis Investigative Site

    RECRUITING

    CABA, Buenos Aires, C1181ACH, Argentina

  • Novartis Investigative Site

    RECRUITING

    CABA, Buenos Aires, C1425BEA, Argentina

  • Novartis Investigative Site

    RECRUITING

    Capital Federal, C1023AAB, Argentina

  • Novartis Investigative Site

    RECRUITING

    Sydney, New South Wales, 2010, Australia

  • Novartis Investigative Site

    RECRUITING

    Sydney, New South Wales, 2031, Australia

  • Novartis Investigative Site

    RECRUITING

    Brisbane, Queensland, 4101, Australia

  • Novartis Investigative Site

    RECRUITING

    Brussels, 1200, Belgium

  • Novartis Investigative Site

    RECRUITING

    Angers, 49933, France

  • Novartis Investigative Site

    RECRUITING

    Bordeaux, 33076, France

  • Novartis Investigative Site

    RECRUITING

    Bron, 69677, France

  • Novartis Investigative Site

    RECRUITING

    Caen, 14033, France

  • Novartis Investigative Site

    RECRUITING

    Dijon, 21000, France

  • Novartis Investigative Site

    RECRUITING

    Lille, 59000, France

  • Novartis Investigative Site

    RECRUITING

    Marseille, 13885, France

  • Novartis Investigative Site

    RECRUITING

    Montpellier, 34295, France

  • Novartis Investigative Site

    RECRUITING

    Paris, 75010, France

  • Novartis Investigative Site

    RECRUITING

    Paris, 75015, France

  • Novartis Investigative Site

    RECRUITING

    Toulouse, 31054, France

  • Novartis Investigative Site

    RECRUITING

    Tours, 37044, France

  • Novartis Investigative Site

    RECRUITING

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Novartis Investigative Site

    WITHDRAWN

    Mannheim, Baden-Wurttemberg, 68305, Germany

  • Novartis Investigative Site

    RECRUITING

    Cologne, North Rhine-Westphalia, 50937, Germany

  • Novartis Investigative Site

    RECRUITING

    Leipzig, Saxony, 04103, Germany

  • Novartis Investigative Site

    RECRUITING

    Berlin, 13353, Germany

  • Novartis Investigative Site

    RECRUITING

    Bologna, BO, 40138, Italy

  • Novartis Investigative Site

    RECRUITING

    Milan, MI, 20122, Italy

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00165, Italy

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00168, Italy

  • Novartis Investigative Site

    RECRUITING

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    RECRUITING

    Naples, 80122, Italy

  • Novartis Investigative Site

    RECRUITING

    Nijmegen, Gelderland, 6500HB, Netherlands

  • Novartis Investigative Site

    RECRUITING

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Novartis Investigative Site

    RECRUITING

    Palma, Balearic Islands, 07120, Spain

  • Novartis Investigative Site

    RECRUITING

    Esplugues, Barcelona, 08950, Spain

  • Novartis Investigative Site

    RECRUITING

    L'Hospitalet de Llobregat, Barcelona, 08907, Spain

  • Novartis Investigative Site

    RECRUITING

    A Coruña, 15006, Spain

  • Novartis Investigative Site

    RECRUITING

    Barcelona, 08035, Spain

  • Novartis Investigative Site

    RECRUITING

    Madrid, 28009, Spain

  • Novartis Investigative Site

    RECRUITING

    Madrid, 28046, Spain

  • Novartis Investigative Site

    RECRUITING

    Lausanne, 1011, Switzerland

  • Oregon Health Science University

    RECRUITING

    Portland, Oregon, 97239, United States

    Contact Email: •••••@•••••

    Contact

  • U of TX Health Science Ct

    RECRUITING

    Houston, Texas, 77030, United States

    Contact

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

  • UCSF Benioff Children s Hospital

    RECRUITING

    Oakland, California, 94609, United States

    Contact

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

  • Univ Hospital Of Cleveland

    RECRUITING

    Cleveland, Ohio, 44106, United States

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

    Contact

  • WA Uni School Of Med

    RECRUITING

    St Louis, Missouri, 63110, United States

    Contact Email: •••••@•••••

    Contact

Conditions

Explore the condition pages connected to this study.

LYMPHATIC MALFORMATIONS LYMPHATIC MALFORMATIONS