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Tofersen for Non-SOD1 ALS

This study is looking at whether tofersen is safe and effective for adults with Amyotrophic Lateral Sclerosis (ALS) that is not caused by a SOD1 gene mutation. Tofersen is already approved for a different type of ALS (SOD1-ALS). Researchers want to see if tofersen can lower levels of neurofilament light chain (NfL), a marker of nerve damage, in your blood and spinal fluid. They will also check if it's safe and if it helps with your symptoms and quality of life. You would receive 100 mg of tofersen through a lumbar puncture (spinal tap) eight times over 24 weeks. The study aims to enroll 30 participants and is currently recruiting.

Study design
This is an interventional study with a planned enrollment of 30 participants. The phase of the study is not specified.
What's involved
You would receive 100 mg of tofersen via lumbar puncture eight times over 24 weeks, at specific time points.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint measures changes from baseline to Week 28, suggesting follow-up for at least 28 weeks.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07294144

Tofersen in Non-SOD1 ALS

Recruiting
PHASE2Ages 18+InterventionalTreatment
Washington University School of Medicine
~30 participants
Updated 2026-06-25 on ClinicalTrials.gov
What's tested:Tofersen

At a glance

Recruiting sites
3 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Proportion of participants with ≥30% reduction in plasma NfL
Measured over From Baseline to Week 28

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Explore related trials

Amyotrophic Lateral Sclerosis

NCT07294144

Where you'd take part

This study runs at 3 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Emory University

    Atlanta, Georgiastudy coordinator listed

    Recruiting

  • Massachusetts General Hospital

    Boston, Massachusettsstudy coordinator listed

    Recruiting

  • Washington University ALS Center

    St Louis, Missouristudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Karon Simmons
[email protected]
404-712-4182
Email the study team

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Eligibility criteria

Inclusion

Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use PHI in accordance with national and local participant privacy regulations.
Aged 18 years or older at the time of informed consent.
Confirmed diagnosis of ALS.
Time since onset of weakness due to ALS ≤ 24 months at the time of the screening visit.
Prior confirmed genetic testing negative for SOD1 and FUS mutations. Participants with mutations in genes other than SOD1 and FUS may be enrolled at the discretion of the Site Investigator.
SVC ≥ 50% of predicted value as adjusted for sex, age, and height (from the sitting position).
Medically able to undergo the study procedures and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.
All participants must agree to practice effective contraception during the study and be willing and able to continue contraception for 5 months after their last dose of study treatment.
If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit.
If taking edaravone, participant must have initiated edaravone ≥ 60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study.

Exclusion

Treatment with another investigational drug (including investigational drugs for ALS through compassionate use or expanded access 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.
Current enrollment in any other interventional study.
History of drug abuse or alcoholism within ≤ 6 months of study enrollment that would limit participation in the study, as determined by the Investigator.
Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the screening period.
Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that according to the Investigator would interfere with the conduct or assessments of 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 neurofilament, in the opinion of the Investigator.
Female participants who are pregnant or currently breastfeeding.
Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression ≤ 90 days, as determined by the Investigator.
History of allergies to a broad range of anesthetics.
Tracheostomy.
Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally could place a participant at an increased risk for intraoperative or postoperative bleeding. These could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).
Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication that cannot be safely held before and/or after an LP procedure according to local or institutional guidelines and/or Investigator determination.
Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter.
Clinically significant abnormalities in hematology or clinical chemistry parameters, as determined by the Investigator, which would render the participant unsuitable for enrollment.
Inability to comply with study requirements.
Other unspecified reasons that, in the opinion of the Investigator, make the participant unsuitable for enrollment.

What this trial measures

  • Proportion of participants with ≥30% reduction in plasma NfLFrom Baseline to Week 28