Drug trial halted early for rare genetic disorder in kids

NCT ID NCT05307679

Summary

This study tested an experimental drug called basmisanil in children aged 2 to 14 with Dup15q syndrome, a rare genetic disorder that causes developmental delays, seizures, and learning difficulties. The main goal was to see if the drug could safely improve daily living skills, communication, and behavior over one year of treatment. The trial was terminated early and enrolled only 7 participants, so its results are very limited.

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Contacts and locations

Locations

  • Children's Hospital Los Angeles

    Los Angeles, California, 90010, United States

  • Evelina London Children's Hospital

    London, SE1 7EH, United Kingdom

  • Hospital Sant Joan De Deu

    Esplugues de Llobregas, Barcelona, 08950, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Rush University Medical Center

    Chicago, Illinois, 60612-3244, United States

Conditions

Explore the condition pages connected to this study.