New drug targets root cause of rare overgrowth diseases in kids and adults

NCT ID NCT06789913

First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 30 times

Summary

This study tests a new drug, RLY-2608, designed to block a faulty protein that causes abnormal growth in people with PIK3CA-related overgrowth spectrum (PROS) and related malformations. The trial enrolls 277 adults and children to see if the drug can safely shrink overgrown tissue and improve symptoms. Participants are randomly assigned to receive the drug or a placebo in the final part of the study.

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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: •••••@•••••

Locations

  • A.O.U Città della Salute e della Scienza di Torino

    RECRUITING

    Torino, Italy

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

  • Arkansas Children's Hospital

    RECRUITING

    Little Rock, Arkansas, 72202, United States

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

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

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

  • Children's Health Queensland Hospital and Health

    RECRUITING

    South Brisbane, Queensland, 4101, Australia

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

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

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

  • Children's Hospital of Atlanta

    RECRUITING

    Atlanta, Georgia, 30329, United States

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

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

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

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

  • Cleveland Clinic Children's

    RECRUITING

    Cleveland, Ohio, 44195, United States

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

  • Corewell Health

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

    Contact Email: •••••@•••••

  • Great Ormond Street Hospital for Children

    RECRUITING

    London, United Kingdom

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

  • Hospital Sant Joan de Deu

    RECRUITING

    Barcelona, Spain

    Contact Email: •••••@•••••

    Contact Email: •••••@•••••

  • Hospital Universitario La Paz

    RECRUITING

    Madrid, Spain

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

  • Johns Hopkins Medical Institute

    RECRUITING

    Baltimore, Maryland, 21287, United States

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

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

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

  • Monash Health

    RECRUITING

    Clayton, Victoria, 3168, Australia

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

  • Murdoch Children's Research Institute

    RECRUITING

    Parkville, Victoria, 3052, Australia

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

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

  • Ospedale Pediatrico Bambino Gesù IRCCS

    RECRUITING

    Roma, Italy

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

  • Phoenix Children's Hospital

    RECRUITING

    Phoenix, Arizona, 85016, United States

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

  • Riley Children's Hospital

    RECRUITING

    Indianapolis, Indiana, 46202, United States

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

  • Royal Melbourne Hospital

    RECRUITING

    Parkville, Victoria, 3050, Australia

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

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98101, United States

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

  • Stanford University

    RECRUITING

    Palo Alto, California, 94304, United States

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

  • Sydney Children's Hospital, Randwick

    RECRUITING

    Randwick, New South Wales, 2031, Australia

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

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

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

  • UC Louvain

    RECRUITING

    Ottignies-Louvain-la-Neuve, Belgium

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

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

  • UNC Chapel Hill

    RECRUITING

    Chapel Hill, North Carolina, 27599, United States

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

  • University of California, Los Angeles

    RECRUITING

    Los Angeles, California, 90095, United States

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

  • University of California, San Francisco

    RECRUITING

    San Francisco, California, 94158, United States

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

    Contact Email: •••••@•••••

  • University of Halle

    RECRUITING

    Halle, Germany, Germany

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

  • University of Wisconsin, Madison

    RECRUITING

    Madison, Wisconsin, 53715, United States

    Contact Email: •••••@•••••

  • Washington University School of Medicine

    RECRUITING

    St Louis, Missouri, 63110, United States

    Contact Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.