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New hope for ALS: first human trial of Gene-Targeting drug begins

NCT ID NCT06351592

Recruiting now Disease control Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 23, 2026 · Updated 2 times

Summary

This study tests an experimental drug called ALN-SOD in people with ALS caused by a mutation in the SOD1 gene. The drug is given via spinal injection to target the root cause of the disease. The main goal is to check safety, but researchers will also measure effects on biomarkers and symptoms. 54 participants are being recruited for this early-stage trial.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
ALN-SOD
What this could lead to
If it works, this could slow or stop the progression of ALS in people with SOD1 mutations, potentially preserving function and extending life.
What could go wrong
This is a very early (first-in-human) trial with only 54 participants, so safety and effectiveness are not yet known. The drug may not work or could cause unexpected side effects.

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 1 familial amyotrophic lateral sclerosis

As listed by the trial registrant

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

AMYOTROPHIC LATERAL SCLEROSIS (ALS) MUTATION IN THE SUPEROXIDE DISMUTASE-1 (SOD1) GENE

Contacts and locations

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

  • MTZ Clinical Research powered by Pratia

    RECRUITING

    Warsaw, 02-172, Poland

  • Macquarie University

    RECRUITING

    Sydney, New South Wales, 2109, Australia

  • Medical University of Silesia

    RECRUITING

    Zabrze, Silesian Voivodeship, 41-800, Poland

  • Montreal Neurological Institute and Hospital

    RECRUITING

    Montreal, Quebec, H3A 2B4, Canada

  • Noorlands University Hospital-Department of Neurology

    RECRUITING

    Umeå, Noorland, 907 37, Sweden

  • 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

  • Universitaetsklinikum Ulm AoR

    RECRUITING

    Ulm, Baden-Wurttemberg, 89081, Germany

  • 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

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