Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)

First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations

First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS) (NCT06351592) is a Phase 1 / Phase 2 interventional studying Amyotrophic Lateral Sclerosis (ALS) and Mutation in the Superoxide Dismutase-1 (SOD1) Gene, sponsored by Regeneron Pharmaceuticals. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people. The aim of the study is to see how safe and tolerable the study drug is. The study is looking at several other research questions, including: * The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as Cerebrospinal Fluid (CSF) * How much study drug is in the blood and in the CSF, at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects) * What effects the study drug has on ALS symptoms

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Amyotrophic Lateral Sclerosis (ALS), a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 42 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Key Who May Qualify: 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 Who Should NOT Join This Trial: 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 Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

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

Treatments Being Tested

DRUG

ALN-SOD

Administered per the protocol

OTHER

Diluent

Administered per the protocol

DRUG

Placebo (PB)

Administered per the protocol

Locations (16)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Concord Repatriation General Hospital

Concord, New South Wales, Australia

Macquarie University

Sydney, New South Wales, Australia

Sunshine Coast University Hospital

Birtinya, Queensland, Australia

KU Leuven

Leuven, Vlaams-Brabant, Belgium

University of Alberta Hospital, Edmonton, Division of Neurology

Edmonton, Alberta, Canada

University Hospital - London Health Sciences Centre

London, Ontario, Canada

Sunnybrook Research Institute

Toronto, Ontario, Canada

Montreal Neurological Institute and Hospital

Montreal, Quebec, Canada

Hokkaido University Hospital

Sapporo, Hokkaido, Japan

Tokushima University Hospital

Tokushima, Tokushima, Japan

Toho University Omori Medical Center

Ōta-ku, Tokyo, Japan

Kyoto University Hospital

Kyoto, Japan

Hanyang University Seoul Hospital

Seoul, South Korea

Seoul National University Hospital

Seoul, South Korea

Kaohsiung Chang Gung Memorial Hospital

Kaohsiung City, Taiwan

Taipei Veterans General Hospital

Taipei, Taiwan

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06351592), the sponsor (Regeneron Pharmaceuticals), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06351592 clinical trial studying?

This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people. The aim of the study is to see how safe and tolerable the study drug is. The study is looking at several other research questions, including: * The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surround… The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06351592?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06351592?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06351592. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06351592. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.