First human test of gene therapy for inherited dementia begins

NCT ID NCT04408625

Summary

This is an early-stage study testing a new gene therapy called LY3884963 for people with a specific genetic form of frontotemporal dementia (FTD). The main goals are to check if the treatment is safe and to see if it can increase levels of a protective protein in the brain and spinal fluid that is low in these patients. The study will follow 35 participants for up to 5 years to monitor their health and track changes in disease-related markers.

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Contacts and locations

Locations

  • AP-HM Hôpital de La Timone

    Saint-Pierre, Marseille, 13386, France

  • Centre Mémoire de Ressources

    Lille, 59000, France

  • Hospital Clinic de Barcelona, Villaroel 170 Servicio de Neurología

    Barcelona, 08036, Spain

  • Hospital Universitario de Donostia, Servicio De Neurologia, Consultas Externas Neurologia, San Sebastian, Guipúzcoa

    Donostia / San Sebastian, 20014, Spain

  • Hospital of the University of Pennsylvania, 3 West Gates Building, 3400 Spruce Street

    Philadelphia, Pennsylvania, 19104, United States

  • Lahey Hospital & Medical Center, 41 Burlington Mall Road

    Burlington, Massachusetts, 01805, United States

  • Le Ber, Institut du Cerveau et de la Moelle Epinière

    Paris, 75013, France

  • PPD Phase 1 Clinic, 100 West Gore Street, Suite 202

    Orlando, Florida, 32806, United States

  • Royal Prince Alfred Hospital, Brain & Mind Research Institute, 94 Mallet Street

    Camperdown, New South Wales, 2050, Australia

  • UZ Leuven, Neurologie Herestraat 49

    Leuven, 3000, Belgium

  • University College London,Queen Square, Dementia Research Building, London,

    London, WC1N 3BG, United Kingdom

  • k2 Medical Research-Maitland

    Maitland, Florida, 32751-5669, United States

Conditions

Explore the condition pages connected to this study.