Can a new drug stop ALS before it starts? major trial underway
NCT ID NCT04856982
First seen Apr 02, 2026 · Last updated May 20, 2026 · Updated 10 times
Summary
This phase 3 study tests whether the drug tofersen can delay or prevent the start of ALS in adults who carry a SOD1 gene mutation but do not yet have symptoms. The trial includes 158 participants and compares tofersen to a placebo. Researchers will measure how many people develop ALS within two years and track safety and disease markers.
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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.
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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Austin Neuromuscular Center
Austin, Texas, 78756, United States
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Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Torino, 10124, Italy
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California Pacific Medical Center Research Institute
San Francisco, California, 94107, United States
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Centrum Medyczne NeuroProtect
Warsaw, 01-684, Poland
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Columbia University Medical center
New York, New York, 10032, United States
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Genge Partners
Montreal, Quebec, H4A 3T2, Canada
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Groupe Hospitalier Pitie-Salpetriere
Paris, Paris, 75651, France
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Hanyang University Seoul Hospital
Seoul, 04763, South Korea
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Holy Cross Hospital
Fort Lauderdale, Florida, 33308, United States
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HonorHealth Neurology
Scottsdale, Arizona, 85258, United States
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Hospital Sao Paulo
São Paulo, São Paulo, 04037-002, Brazil
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Hospital Universitari i Politecnic La Fe
Valencia, 46026, Spain
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Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
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Kagoshima University Hospital
Kagoshima, Kagoshima-ken, 890-8520, Japan
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Macquarie University Hospital
Macquarie Park, New South Wales, 2109, Australia
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Massachusetts General Hospital, MA
Charlestown, Massachusetts, 02129, United States
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Medizinische Hochschule Hannover
Hanover, Lower Saxony, 30625, Germany
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NeuroProtect Sp. z o.o.
Warsaw, Masovian Voivodeship, 01-684, Poland
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Norrlands Universitetssjukhus
Umeå, 90185, Sweden
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Northwestern Medicine
Chicago, Illinois, 60611, United States
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PSEG Centro de Pesquisa Clinica
São Paulo, 04038-002, Brazil
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Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5, Canada
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The Emory Clinic
Atlanta, Georgia, 30322-4200, United States
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UZ Leuven
Leuven, 3000, Belgium
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Universitaetsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081, Germany
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University Hospital of Umea
Umeå, Västerbotten County, 90185, Sweden
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University of Calgary
Calgary, Alberta, T2N4Z6, Canada
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University of California San Diego Medical Center
La Jolla, California, 92093-0949, United States
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University of Miami School of Medicine
Miami, Florida, 33136, United States
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University of Sheffield
Sheffield, South Yorkshire, S10 2RX, United Kingdom
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University of Tokyo Hospital
Bunkyō City, Tokyo-To, 113-8655, Japan
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.