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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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158 people
The number who actually took part.
- Started
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May 2021
- Expected to finish
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Apr 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Key Part A Inclusion Criteria: * Participants should have a protocol-defined rapidly progressive SOD1 mutation, confirmed by a central reader, or a SOD1 mutation that is approved for inclusion by an external mutation adjudication committee. * Participants with plasma NfL level less than the protocol-defined threshold. * Participants who are clinically presymptomatic for ALS (i.e., must not have clinically manifest ALS). Key Part A Exclusion Criteria: * History or positive test result at screening for human immunodeficiency virus (HIV). The requirement for testing at Screening may be omitted if it is not permitted by local regulations. * Current hepatitis C infection (defined as positive Hepatitis C Virus (HCV) antibody and detectable HCV RNA). Participants with positive HCV antibody and undetectable HCV Ribonucleic Acid (RNA) are eligible to participate in the study (United States Centers for Disease Control and Prevention). * Current hepatitis B infection (defined as positive for hepatitis B surface antigen (HBsAg) and/or anti-Hepatitis B Core antibody (HBc)). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive anti-HBc, and positive anti-hepatitis B surface antibody (HBs) or vaccination (defined as negative HBsAg, negative anti-HBc, and positive anti- HBs) are eligible to participate in the study. * History of systemic hypersensitivity reaction to tofersen, the excipients contained in the formulation, and if appropriate, any diagnostic agents to be administered during the study. * History of confounding neuromuscular or neurological disorder that is expected to have a progressive (i.e., worsening) course during the study, and/or is expected to be associated with elevations in NF, in the opinion of the Investigator. * Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that if not managed optimally could place a participant at an increased risk for intraoperative or postoperative bleeding. * Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression ≤ 90 days of screening, which in the opinion of the Investigator would interfere with the study procedures. * Treatment with riluzole, edaravone, and/or sodium phenylbutyrate/taurursodiol (also known as ursodoxicoltaurine). If the participant has been on riluzole, edaravone, and/or sodium phenylbutyrate/taurursodiol, the medication(s) must be discontinued for at least 5 half-lives prior to Screening. * Use of off-label treatments for ALS. * Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed. * Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication (e.g., clopidogrel) that cannot be safely continued or held for an LP procedure, if necessary, according to local or institutional guidelines and/or Investigator determination. * Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment, biological agent, device, or approved therapy for investigational use. Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator. NOTE: Other protocol defined Inclusion/Exclusion criteria will apply.
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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
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