Brain drug tested for rare genetic disorder in kids

NCT ID NCT05630066

Summary

This study tested a drug called alogabat in children and teenagers with Angelman syndrome, a rare genetic disorder. The main goals were to see how the drug moves through the body, if it is safe, and if it changes brain wave patterns. Forty-eight participants aged 5 to 17 took the drug daily for 12 weeks while researchers monitored them.

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Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHRU de Brest

    Brest, 29609, France

  • CHU Dijon Bourgogne Hôpital François Mitterand

    Dijon, 21000, France

  • Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine

    Carrboro, North Carolina, 27510, United States

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Corporacio Sanitaria Parc Tauli

    Sabadell, Barcelona, 08208, Spain

  • Dr. Von Haunersches Kinderspital

    München, 80337, Germany

  • Groupe Hospitalier Necker Enfants Malades

    Paris, 75015, France

  • Hopital la Timone Enfants

    Marseille, 13005, France

  • Hospital Sant Joan de Deu

    Esplugues de Llobregat · Barcelona, Barcelona, 08950, Spain

  • Hospital Universitario Puerta De Hierro Majadahonda

    Madrid, 28222, Spain

  • Hospital Universitario de Navarra;Unidad de Neuropediatría

    Pamploa, Navarre, 31008, Spain

  • IRCCS Eugenio Medea

    Conegliano Veneto (TV), Veneto, 31015, Italy

  • IRCCS Istituto G. Gaslini

    Genoa, Liguria, 16147, Italy

  • Multicare Institute for Research and Innovation

    Tacoma, Washington, 98405, United States

  • Ospedale Pediatrico Bambino Gesù

    Rome, Lazio, 00165, Italy

  • Queensland Children?s Hospital

    South Brisbane, Queensland, 4101, Australia

  • Rush Medical Center

    Chicago, Illinois, 60612, United States

  • Vanderbilt Children's Hospital

    Nashville, Tennessee, 37232-9119, United States

Conditions

Explore the condition pages connected to this study.