First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
NCT ID: NCT06351592
Last Updated: 2025-09-17
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
42 participants
INTERVENTIONAL
2024-08-28
2029-04-26
Brief Summary
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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
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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Cohort 1 - Low Dose
Placebo during double-blind treatment period
ALN-SOD
Administered by intrathecal (IT) injection
Diluent
Administered by IT injection
Placebo (PB)
Administered by IT injection
Cohort 2 - Mid Dose
Placebo during double-blind treatment period
ALN-SOD
Administered by intrathecal (IT) injection
Diluent
Administered by IT injection
Placebo (PB)
Administered by IT injection
Cohort 3 - High Dose
Placebo during double-blind treatment period
ALN-SOD
Administered by intrathecal (IT) injection
Diluent
Administered by IT injection
Placebo (PB)
Administered by IT injection
Cohort 4 (Optional) ≤ High Dose
Placebo during double-blind treatment period
ALN-SOD
Administered by intrathecal (IT) injection
Diluent
Administered by IT injection
Placebo (PB)
Administered by IT injection
Interventions
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ALN-SOD
Administered by intrathecal (IT) injection
Diluent
Administered by IT injection
Placebo (PB)
Administered by IT injection
Eligibility Criteria
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Inclusion Criteria
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
Exclusion Criteria
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
18 Years
ALL
No
Sponsors
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Regeneron Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Management
Role: STUDY_DIRECTOR
Regeneron Pharmaceuticals
Locations
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Concord Repatriation General Hospital
Concord, New South Wales, Australia
Macquarie University
Sydney, New South Wales, Australia
Sunshine Coast University Hospital
Birtinya, Queensland, Australia
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
Taipei Veterans General Hospital
Taipei, , Taiwan
Countries
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Central Contacts
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Other Identifiers
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2023-510344-20-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
ALN-SOD-ALS-2351
Identifier Type: -
Identifier Source: org_study_id
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