One-Shot spinal injection aims to silence deadly ALS gene
NCT ID NCT06100276
Summary
This early-stage study is testing a one-time gene therapy called AMT-162 for adults with ALS caused by a specific faulty SOD1 gene. The therapy is injected into the spinal fluid and aims to 'silence' the harmful gene to slow the disease. Researchers will first check if the treatment is safe and tolerated in a small group of 20 participants, while also looking for early signs that it might help.
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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
Explore the condition pages connected to this study.