Promising Gene-Targeting drug for ALS enters final trial phase
NCT ID NCT04768972
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests an experimental drug called ION363 in people with a rare, inherited form of ALS caused by FUS gene mutations. The goal is to see if the drug can slow the disease and help people live longer. About 89 participants will receive the drug via spinal injection. This is a late-stage (Phase 3) trial, meaning the drug has shown promise in earlier studies.
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Citta della Salute e della Scienza di Torino - Ospedale le Molinette
Torino, 10126, Italy
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Columbia University Medical Center
New York, New York, 10032, United States
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Hanyang University Seoul Hospital
Seoul, 4763, South Korea
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Hospital Universitari de Bellvitge
Barcelona, 8907, Spain
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Johns Hopkins University
Baltimore, Maryland, 21205, United States
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Kantonsspital St. Gallen
Sankt Gallen, 9007, Switzerland
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King's College Hospital
London, SE5 9RT, United Kingdom
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Linden spólka z ograniczona odpowiedzialnoscia spólka komandytow
Krakow, 30-721, Poland
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Montreal Neurological Institute
Montreal, Quebec, H3A 2B4, Canada
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PSEG Centro de Pesquisa Clinica S.A.
São Paulo, 04038-002, Brazil
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Seoul National University Hospital
Seoul, 3080, South Korea
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St. James Hospital
Dublin, D08 A978, Ireland
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Stanford University Medical Center
Palo Alto, California, 94304, United States
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Taipei Veterans General Hospital (VGHTP)
Taipei, 11217, Taiwan
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The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210, United States
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Toho University Omori Medical Center
Tokyo, 143-8541, Japan
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UZ Leuven
Leuven, VL-Brabant, 3000, Belgium
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Universitaetsmedizin Rostock
Rostock, 18147, Germany
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
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University Hospital of Umea
Umeå, SE-901 85, Sweden
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University of California San Diego
La Jolla, California, 92037, United States
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University of Utah
Salt Lake City, Utah, 84132, United States
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Universitätsklinikum Ulm
Ulm, 89081, Germany
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Washington University School of Medicine
St Louis, Missouri, 63110, United States