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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
Led by Regeneron Pharmaceuticals · Updated on 2026-02-27
42
Participants Needed
16
Research Sites
N/A
Total Duration
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AI-Summary
What this Trial Is About
Researchers are evaluating an experimental drug called ALN-SOD for people with Amyotrophic Lateral Sclerosis (ALS) caused by changes in the Superoxide Dismutase-1 (SOD1) gene, known as SOD1-ALS. This is the first time ALN-SOD will be given to people, aiming to assess its safety and how well it is tolerated. The study also looks at how the drug affects specific biomarkers in blood and cerebrospinal fluid, the presence of antibodies against the drug, and its impact on ALS symptoms. Participants will receive ALN-SOD in multiple ascending doses administered intrathecally, meaning directly into the fluid around the spinal cord. The study includes several groups receiving different dose levels, including an optional cohort with a dose up to a high level. There is also a 4-week double-blind period where some participants receive a placebo. The trial lasts up to approximately 228 weeks, covering treatment and follow-up phases. During the study, participants will undergo regular assessments including blood and cerebrospinal fluid tests to measure drug levels and biomarkers like neurofilament light chain and SOD1 protein. Researchers will monitor adverse events and check for antibodies that might affect the drug's action. They will also track changes in ALS symptoms using standard scales. The total participation may last over four years, with ongoing safety and effect evaluations throughout this period.
CONDITIONS
Brief Title
First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Weakness caused by ALS with a SOD1 gene variant linked or likely linked to ALS
- Slow Vital Capacity (SVC) at least 50% of predicted value for age, gender, and height, measured upright
- Body Mass Index (BMI) of 35 kg/m2 or less at screening
- Stable dose of riluzole or edaravone for at least 4 weeks before dosing, if taking these medications
- Platelet count greater than 50,000 per microliter
- Normal blood pressure readings as defined in the protocol
You will not qualify if you...
- Currently participating in another interventional clinical trial
- Has had a tracheostomy
- Has dementia as assessed by the investigator
- Has uncontrolled psychiatric illness including psychosis, recent suicidal thoughts, or untreated major depression in past 30 days
- History of brain or spinal disease/injury interfering with lumbar puncture, cerebrospinal fluid circulation, or safety assessment
- Has an implanted shunt or central nervous system catheter
- Has any condition that may confuse study results or increase risk as judged by the investigator
- Hospitalized for more than 24 hours for any reason other than ALS within 30 days before screening
- Received treatment with tofersen within 6 months before screening
- Other protocol-defined inclusion or exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 4 weeks (double-blind treatment period)
Participants receive multiple ascending doses of ALN-SOD or placebo administered intrathecally as part of the study.
Weekly visits for up to 4 weeks
Duration - Up to approximately week 228
Participants are monitored for safety, tolerability, and biomarker changes after treatment ends.
Periodic visits up to week 228
Trial Site Locations
Total: 16 locations
1
Concord Repatriation General Hospital
Concord, New South Wales, Australia, 2139
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2
Macquarie University
Sydney, New South Wales, Australia, 2109
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3
Sunshine Coast University Hospital
Birtinya, Queensland, Australia, 4575
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4
KU Leuven
Leuven, Vlaams-Brabant, Belgium, 3000
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5
University of Alberta Hospital, Edmonton, Division of Neurology
Edmonton, Alberta, Canada, T6G 2G3
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6
University Hospital - London Health Sciences Centre
London, Ontario, Canada, N6A 5A5
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7
Sunnybrook Research Institute
Toronto, Ontario, Canada, M4N 3M5
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8
Montreal Neurological Institute and Hospital
Montreal, Quebec, Canada, H3A 2B4
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9
Hokkaido University Hospital
Sapporo, Hokkaido, Japan, 060-8648
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10
Tokushima University Hospital
Tokushima, Tokushima, Japan, 770-0042
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11
Toho University Omori Medical Center
Ōta-ku, Tokyo, Japan, 143-8541
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12
Kyoto University Hospital
Kyoto, Japan, 606-8507
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13
Hanyang University Seoul Hospital
Seoul, South Korea, 04763
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14
Seoul National University Hospital
Seoul, South Korea, 3080
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15
Kaohsiung Chang Gung Memorial Hospital
Kaohsiung City, Taiwan, 83301
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16
Taipei Veterans General Hospital
Taipei, Taiwan, 11211
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Research Team
C
Clinical Trials Administrator
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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