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New hope for ALS: first human trial of Gene-Targeting drug begins

NCT ID NCT06351592

Disease control Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 11, 2026 · Updated 3 times

Summary

This study tests an experimental drug called ALN-SOD in people with ALS caused by a mutation in the SOD1 gene. The drug is given via spinal injection to target the root cause of the disease. The main goal is to check safety, but researchers will also measure effects on biomarkers and symptoms. 54 participants are being recruited for this early-stage trial.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ALN-SOD
What this could lead to
If it works, this could slow or stop the progression of ALS in people with SOD1 mutations, potentially preserving function and extending life.
What could go wrong
This is a very early (first-in-human) trial with only 54 participants, so safety and effectiveness are not yet known. The drug may not work or could cause unexpected side effects.

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

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 54 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2024

Expected to finish

Mar 2032

An estimate. End dates often move.

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: 1. Weakness attributable to ALS and a SOD1 variant that has been previously described as associated with ALS or is considered likely to cause ALS, as defined in the protocol 2. Slow Vital Capacity (SVC) ≥50% predicted value based on age, gender and height, measured in upright position 3. Body Mass Index (BMI) ≤35 kg/m2 at time of screening 4. If participants are taking riluzole or edaravone, they must be on a stable dose for at least 4 weeks prior to initial dosing visit and are expected to remain at that dose until the end of the study 5. Platelet count \>50,000/microliter 6. Has normal blood pressure readings, as defined in the protocol Key Exclusion Criteria: 1. Concurrent participation in another interventional clinical trial 2. Has had a tracheostomy 3. Has dementia, as assessed by the investigator 4. Has uncontrolled psychiatric disease, including psychosis, active or recent suicidal ideation, untreated major depression, in the past 30 days 5. Has a medical history of brain or spinal disease/injury that would interfere with the Lumbar Puncture (LP) process, CSF circulation or safety assessment, as defined in the protocol 6. Presence of an implanted shunt for the drainage of CSF or an implanted Central Nervous System (CNS) catheter 7. Presents any concern to the study investigator that might confound the results of the study or poses an additional risk to the participant by their participation in the study 8. Was hospitalized (ie, \>24 hours) for any reason other than ALS within 30 days of screening 9. Has received treatment with tofersen within 6 months prior to screening NOTE: Other protocol defined inclusion / exclusion criteria apply

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis amyotrophic lateral sclerosis type 1 familial amyotrophic lateral sclerosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Amyotrophic lateral sclerosis (ALS) Mutation in the superoxide dismutase-1 (SOD1) gene

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    22 sites in 9 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Concord Repatriation General Hospital

    RECRUITING

    Concord, New South Wales, 2139, Australia

  • Hanyang University Seoul Hospital

    RECRUITING

    Seoul, 04763, South Korea

  • Hokkaido University Hospital

    RECRUITING

    Sapporo, Hokkaido, 060-8648, Japan

  • KU Leuven

    RECRUITING

    Leuven, Vlaams-Brabant, 3000, Belgium

  • Kaohsiung Chang Gung Memorial Hospital

    RECRUITING

    Kaohsiung City, 83301, Taiwan

  • Karolinska University Hospital, Karolinska Institutet-ALS Center-Department of Clinical Neuroscience

    RECRUITING

    Huddinge, Stockholm County, 141 57, Sweden

  • Kyoto University Hospital

    RECRUITING

    Kyoto, 606-8507, Japan

  • MTZ Clinical Research powered by Pratia

    RECRUITING

    Warsaw, 02-172, Poland

  • Macquarie University

    RECRUITING

    Sydney, New South Wales, 2109, Australia

  • Medical University of Silesia

    RECRUITING

    Zabrze, Silesian Voivodeship, 41-800, Poland

  • Montreal Neurological Institute and Hospital

    RECRUITING

    Montreal, Quebec, H3A 2B4, Canada

  • Noorlands University Hospital-Department of Neurology

    RECRUITING

    Umeå, Noorland, 907 37, Sweden

  • Seoul National University Hospital

    RECRUITING

    Seoul, 3080, South Korea

  • Sunnybrook Research Institute

    RECRUITING

    Toronto, Ontario, M4N 3M5, Canada

  • Sunshine Coast University Hospital

    RECRUITING

    Birtinya, Queensland, 4575, Australia

  • Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Neurologii

    RECRUITING

    Krakow, Lesser Poland Voivodeship, 30-688, Poland

  • Taipei Veterans General Hospital

    RECRUITING

    Taipei, 11211, Taiwan

  • Toho University Omori Medical Center

    RECRUITING

    Ōta-ku, Tokyo, 143-8541, Japan

  • Tokushima University Hospital

    RECRUITING

    Tokushima, Tokushima, 770-0042, Japan

  • Universitaetsklinikum Ulm AoR

    RECRUITING

    Ulm, Baden-Wurttemberg, 89081, Germany

  • University Hospital - London Health Sciences Centre

    RECRUITING

    London, Ontario, N6A 5A5, Canada

  • University of Alberta Hospital, Edmonton, Division of Neurology

    RECRUITING

    Edmonton, Alberta, T6G 2G3, Canada

More trials for these conditions

Other studies related to the condition(s) this trial covers.