New gene therapy targets rare, aggressive form of ALS
NCT ID NCT06100276
First seen Oct 31, 2025 · Last updated May 16, 2026 · Updated 26 times
Summary
This study tests a single-dose gene therapy called AMT-162 in 20 adults with a specific genetic form of ALS (SOD1-ALS). The therapy is injected into the spinal fluid to deliver a working gene. The main goal is to check safety and tolerability, while also looking for early signs of benefit. Participants must have a confirmed SOD1 mutation, a certain level of physical function, and adequate lung capacity.
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the original study
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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.
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
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Locations
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Barrow Neurological Institute
Phoenix, Arizona, 85013, United States
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California Pacific Medical Center
San Francisco, California, 94109, United States
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Columbia University Irving Medical Center
New York, New York, 10032, United States
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Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research
Boston, Massachusetts, 02114, United States
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Mayo Clinic Florida
Jacksonville, Florida, 32224, United States
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Mayo Clinic Rochester
Rochester, Minnesota, 55905, United States
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Norrlands Universitetssjukhus
Umeå, Vasterbottens Ian, Sweden
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Northwestern University Feinberg School of Medicine
Chicago, Illinois, 60611, United States
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University of California Irvine
Irvine, California, 92697, United States
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University of Kansas Medical Center
Fairway, Kansas, 66205, United States
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University of Pennsylvania School of Medicine
Philadelphia, Pennsylvania, 19104, United States
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Winship Cancer Institute of Emory University
Atlanta, Georgia, 30322, United States
Conditions
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