Spinal injection trial offers new hope for angelman syndrome

NCT ID NCT05127226

Summary

This study is testing a new drug called ION582 for Angelman syndrome, a rare genetic disorder. The drug is given as an injection into the spinal fluid. The main goals are to see if it's safe and how the body processes it in about 70 participants, including children under 2 years old.

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

  • Azienda Ospedaliera Universitaria Pisana

    RECRUITING

    Pisa, 56126, Italy

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02215, United States

  • Colorado Children's Hospital Research Institute

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Necker-Enfants Malades Hospital

    RECRUITING

    Paris, 75015, France

  • Rady Children's Hospital

    RECRUITING

    San Diego, California, 92123, United States

  • Rush University Medical Center

    RECRUITING

    Chicago, Illinois, 60612, United States

  • STRONG Group University of Oxford

    ACTIVE_NOT_RECRUITING

    Oxford, Oxfordshire, OX3 9DU, United Kingdom

  • Sheba Medical Center

    RECRUITING

    Ramat Gan, 5262100, Israel

  • Sydney Children's Hospital, Kids Cancer Centre

    ACTIVE_NOT_RECRUITING

    Randwick, NSW 2031, Australia

  • Texas Children's Hospital

    ACTIVE_NOT_RECRUITING

    Houston, Texas, 77030, United States

  • University of North Carolina at Chapel Hill School of Medicine

    RECRUITING

    Carrboro, North Carolina, 27510, United States

Conditions

Explore the condition pages connected to this study.