New drug trial targets genetic cause of ALS

NCT ID NCT04768972

Summary

This study is testing an experimental drug called ION363 for people with ALS caused by specific mutations in the FUS gene. The drug is delivered via spinal injection and aims to slow the loss of physical function and improve survival. The trial will involve about 95 participants and includes an initial blinded phase followed by a longer open-label treatment period.

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Contacts and locations

Locations

  • Citta della Salute e della Scienza di Torino - Ospedale le Molinette

    Torino, 10126, Italy

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Hanyang University Seoul Hospital

    Seoul, 4763, South Korea

  • Hospital Universitari de Bellvitge

    Barcelona, 8907, Spain

  • Johns Hopkins University

    Baltimore, Maryland, 21205, United States

  • Kantonsspital St. Gallen

    Sankt Gallen, 9007, Switzerland

  • King's College Hospital

    London, SE5 9RT, United Kingdom

  • Linden spólka z ograniczona odpowiedzialnoscia spólka komandytow

    Krakow, 30-721, Poland

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Montreal Neurological Institute

    Montreal, Quebec, H3A 2B4, Canada

  • PSEG Centro de Pesquisa Clinica S.A.

    São Paulo, 04038-002, Brazil

  • Seoul National University Hospital

    Seoul, 3080, South Korea

  • St. James Hospital

    Dublin, D08 A978, Ireland

  • Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • Taipei Veterans General Hospital (VGHTP)

    Taipei, 11217, Taiwan

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210, United States

  • Toho University Omori Medical Center

    Tokyo, 143-8541, Japan

  • UZ Leuven

    Leuven, VL-Brabant, 3000, Belgium

  • Universitaetsmedizin Rostock

    Rostock, 18147, Germany

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX, Netherlands

  • University Hospital of Umea

    Umeå, SE-901 85, Sweden

  • University of California San Diego

    La Jolla, California, 92037, United States

  • University of Utah

    Salt Lake City, Utah, 84132, United States

  • Universitätsklinikum Ulm

    Ulm, 89081, Germany

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.