New drug trial offers hope for angelman syndrome

NCT ID NCT07157254

Summary

This study is testing an experimental drug called GTX-102 in people with Angelman syndrome, a rare genetic disorder that affects development and causes seizures and movement problems. Researchers want to see if the drug is safe and if it can improve thinking, communication, behavior, and motor skills. The trial includes 60 children and adults, divided into groups based on their age and specific genetic type of Angelman syndrome.

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

  • Akron Children's Hospital

    RECRUITING

    Akron, Ohio, 44308, United States

  • Azienda Ospedaliera Universitaria Meyer IRCCS

    RECRUITING

    Florence, Italy

  • Carum Research Inc.

    RECRUITING

    Dallas, Texas, 75243, United States

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Los Angeles, California, 90048, United States

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Baltimore, Maryland, 21205, United States

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Kansas City, Missouri, 64108, United States

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Pilar, Buenos Aires, Argentina

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Curitiba, Paraná, Brazil

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Santa Cecília, Porto Alegre, Brazil

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Marseille, France

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Paris, France

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Ramat Gan, Israel

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Rome, Italy

  • Clinical Trial Site

    NOT_YET_RECRUITING

    London, United Kingdom

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Oxford, United Kingdom

  • Fondazione IRCCS Istituto Neurologico C. Besta

    RECRUITING

    Milan, Italy

  • Hospital Santa Joao

    RECRUITING

    Porto, Portugal

  • Hospital de Santa Maria

    RECRUITING

    Lisbon, Portugal

  • Rare Disease Research

    RECRUITING

    Hillsborough, North Carolina, 27278, United States

  • Rush University Medical Center

    RECRUITING

    Chicago, Illinois, 60612, United States

  • UT Health Austin

    RECRUITING

    Austin, Texas, 78723, United States

Conditions

Explore the condition pages connected to this study.