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First human trial launches for targeted ALS therapy

NCT ID NCT06351592

First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 28 times

Summary

This study tests an experimental drug called ALN-SOD in people with ALS caused by a specific gene change (SOD1 mutation). The main goal is to check safety and how well the drug is tolerated. Researchers will also measure drug levels in blood and spinal fluid, and look at biomarkers and symptoms. About 42 participants will receive multiple doses of the drug injected into the spine.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Concord Repatriation General Hospital

    RECRUITING

    Concord, New South Wales, 2139, Australia

  • Hanyang University Seoul Hospital

    RECRUITING

    Seoul, 04763, South Korea

  • Hokkaido University Hospital

    RECRUITING

    Sapporo, Hokkaido, 060-8648, Japan

  • KU Leuven

    RECRUITING

    Leuven, Vlaams-Brabant, 3000, Belgium

  • Kaohsiung Chang Gung Memorial Hospital

    RECRUITING

    Kaohsiung City, 83301, Taiwan

  • Kyoto University Hospital

    RECRUITING

    Kyoto, 606-8507, Japan

  • Macquarie University

    RECRUITING

    Sydney, New South Wales, 2109, Australia

  • Montreal Neurological Institute and Hospital

    RECRUITING

    Montreal, Quebec, H3A 2B4, Canada

  • Seoul National University Hospital

    RECRUITING

    Seoul, 3080, South Korea

  • Sunnybrook Research Institute

    RECRUITING

    Toronto, Ontario, M4N 3M5, Canada

  • Sunshine Coast University Hospital

    RECRUITING

    Birtinya, Queensland, 4575, Australia

  • Taipei Veterans General Hospital

    RECRUITING

    Taipei, 11211, Taiwan

  • Toho University Omori Medical Center

    RECRUITING

    Ōta-ku, Tokyo, 143-8541, Japan

  • Tokushima University Hospital

    RECRUITING

    Tokushima, Tokushima, 770-0042, Japan

  • University Hospital - London Health Sciences Centre

    RECRUITING

    London, Ontario, N6A 5A5, Canada

  • University of Alberta Hospital, Edmonton, Division of Neurology

    RECRUITING

    Edmonton, Alberta, T6G 2G3, Canada

Conditions

Explore the condition pages connected to this study.