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Promising Gene-Targeting drug for ALS enters final trial phase

NCT ID NCT04768972

Ongoing Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 22, 2026 · Updated 2 times

Summary

This study tests an experimental drug called ION363 in people with a rare, inherited form of ALS caused by FUS gene mutations. The goal is to see if the drug can slow the disease and help people live longer. About 89 participants will receive the drug via spinal injection. This is a late-stage (Phase 3) trial, meaning the drug has shown promise in earlier studies.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis amyotrophic lateral sclerosis type 6

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AMYOTROPHIC LATERAL SCLEROSIS

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

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