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New gene therapy targets rare, aggressive ALS

NCT ID NCT06100276

First seen Oct 31, 2025 · Last updated May 23, 2026 · Updated 28 times

Summary

This study tests a gene therapy called AMT-162 in 20 adults with a specific genetic form of ALS (SOD1-ALS). The treatment is given as a single injection into the spinal fluid. The main goal is to check safety and tolerability, while also looking for early signs that it might slow the disease. Participants must have a confirmed SOD1 gene mutation and still have at least moderate physical function.

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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

Locations

  • Barrow Neurological Institute

    Phoenix, Arizona, 85013, United States

  • California Pacific Medical Center

    San Francisco, California, 94109, United States

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224, United States

  • Mayo Clinic Rochester

    Rochester, Minnesota, 55905, United States

  • Norrlands Universitetssjukhus

    Umeå, Vasterbottens Ian, Sweden

  • Northwestern University Feinberg School of Medicine

    Chicago, Illinois, 60611, United States

  • University of California Irvine

    Irvine, California, 92697, United States

  • University of Kansas Medical Center

    Fairway, Kansas, 66205, United States

  • University of Pennsylvania School of Medicine

    Philadelphia, Pennsylvania, 19104, United States

  • Winship Cancer Institute of Emory University

    Atlanta, Georgia, 30322, United States

Conditions

Explore the condition pages connected to this study.