Hope for angelman syndrome: major trial tests drug to unlock speech and learning

NCT ID NCT06914609

First seen Nov 01, 2025 · Last updated May 12, 2026 · Updated 25 times

Summary

This study tests a new medicine called ION582 in 158 children and adults with Angelman syndrome, a genetic disorder causing severe developmental delays. The goal is to see if the drug can improve communication and thinking skills. Participants will receive the drug or a placebo, and their progress will be measured over time.

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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

  • Associazione La Nostra Famiglia - IRCCS Eugenio Medea

    NOT_YET_RECRUITING

    Conegliano, 31015, Italy

  • Azienda Ospedaliero Universitaria Pisana

    NOT_YET_RECRUITING

    Pisa, 56126, Italy

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's Hospital

    RECRUITING

    Vancouver, V6H 3V4, Canada

  • Cedars-Sinai Medical Center

    RECRUITING

    Los Angeles, California, 90048, United States

  • Children's Healthcare of Atlanta

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Mercy

    RECRUITING

    Kansas City, Missouri, 64108, United States

  • Children's National Hospital

    RECRUITING

    Washington D.C., District of Columbia, 20011, United States

  • Colorado Children's Hospital Research Institute

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

    RECRUITING

    Sabadell, 08208, Spain

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    NOT_YET_RECRUITING

    Milan, 20133, Italy

  • Great Ormond Street Hospital for Children - NHS Foundation Trust

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hospital Sant Joan de Deu

    RECRUITING

    Barcelona, 08950, Spain

  • Ichan School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States

  • John Radcliffe Hospital

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

  • KK Women's and Children's Hospital

    RECRUITING

    Singapore, 229899, Singapore

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    RECRUITING

    München, 81377, Germany

  • London Health Science Centre - Children's Hospital

    RECRUITING

    London, Ontario, N6A 5W9, Canada

  • McGill University Health Centre

    RECRUITING

    Montreal, Quebec, H4A 3J1, Canada

  • National Center of Neurology and Psychiatry

    RECRUITING

    Kodaira, Tokyo, 187-8551, Japan

  • Nationwide Children's Hospital

    RECRUITING

    Columbus, Ohio, 43205, United States

  • Nicklaus Children's Hospital

    RECRUITING

    Miami, Florida, 33155, United States

  • Osaka Women's and Children's Hospital

    RECRUITING

    Izumi, Osaka, 594-1101, Japan

  • Ospedale Pediatrico Bambino Gesù

    NOT_YET_RECRUITING

    Roma, 00165, Italy

  • Perth Children's Hospital

    RECRUITING

    Nedlands, 6009, Australia

  • Queensland Children's Hospital

    RECRUITING

    South Brisbane, Queensland, 4101, Australia

  • Rady Children's Hospital

    RECRUITING

    San Diego, California, 92123, United States

  • Rush University Medical Center

    RECRUITING

    Chicago, Illinois, 60612, United States

  • Samsung Medical Center

    RECRUITING

    Seoul, 06351, South Korea

  • Seoul National University Hospital

    RECRUITING

    Seoul, 03080, South Korea

  • Sheba Medical Center

    RECRUITING

    Ramat Gan, 5265601, Israel

  • Sydney Children's Hospital

    RECRUITING

    Randwick, 2031, Australia

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • University of Alberta Hospital

    RECRUITING

    Edmonton, T6G 2B7, Canada

  • University of North Carolina at Chapel Hill School of Medicine

    RECRUITING

    Carrboro, North Carolina, 27510, United States

  • Uniwersyteckie Centrum Kliniczne

    RECRUITING

    Gdansk, 80-952, Poland

  • Vanderbilt Clinical Research Center

    RECRUITING

    Nashville, Tennessee, 37232, United States

Conditions

Explore the condition pages connected to this study.