Can a shot stop ALS before it starts? new trial tests tofersen in gene carriers
NCT ID NCT04856982
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 3 trial tests whether the drug tofersen can delay or prevent ALS in adults who carry a SOD1 gene mutation but have no symptoms yet. About 158 participants will receive either tofersen or a placebo, and researchers will track how many develop ALS within two years. The goal is to see if early treatment can stop the disease before it takes hold.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Tofersen (also called BIIB067 or QALSODY)
What this could lead to
If successful, this could show that starting tofersen before symptoms appear delays or prevents the onset of ALS in people with SOD1 mutations.
What could go wrong
This is an early-stage prevention study in a small, specific genetic group. It may not work for all SOD1 mutations, and long-term safety is still being studied.
Disclaimer
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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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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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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