Actively Recruiting
First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
Led by Regeneron Pharmaceuticals · Updated on 2026-02-27
42
Participants Needed
16
Research Sites
353 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official 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 due to ALS with a known or likely SOD1 gene variant linked to ALS
- Slow Vital Capacity (SVC) of at least 50% predicted value based on age, gender, and height
- 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, expected to continue
- Platelet count greater than 50,000 per microliter
- Normal blood pressure as defined in the study protocol
You will not qualify if you...
- Participation in another interventional clinical trial at the same time
- History of tracheostomy
- Diagnosis of dementia
- Uncontrolled psychiatric conditions including psychosis, recent suicidal thoughts, or untreated major depression within 30 days
- Medical history of brain or spinal disease/injury affecting lumbar puncture or cerebrospinal fluid safety
- Presence of implanted shunt or central nervous system catheter
- Any condition that could affect study results or increase risk as judged by the investigator
- Hospitalization longer than 24 hours for reasons other than ALS in the past 30 days
- 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
Trial Site Locations
Total: 16 locations
1
Concord Repatriation General Hospital
Concord, New South Wales, Australia, 2139
Actively Recruiting
2
Macquarie University
Sydney, New South Wales, Australia, 2109
Actively Recruiting
3
Sunshine Coast University Hospital
Birtinya, Queensland, Australia, 4575
Actively Recruiting
4
KU Leuven
Leuven, Vlaams-Brabant, Belgium, 3000
Actively Recruiting
5
University of Alberta Hospital, Edmonton, Division of Neurology
Edmonton, Alberta, Canada, T6G 2G3
Actively Recruiting
6
University Hospital - London Health Sciences Centre
London, Ontario, Canada, N6A 5A5
Actively Recruiting
7
Sunnybrook Research Institute
Toronto, Ontario, Canada, M4N 3M5
Actively Recruiting
8
Montreal Neurological Institute and Hospital
Montreal, Quebec, Canada, H3A 2B4
Actively Recruiting
9
Hokkaido University Hospital
Sapporo, Hokkaido, Japan, 060-8648
Actively Recruiting
10
Tokushima University Hospital
Tokushima, Tokushima, Japan, 770-0042
Actively Recruiting
11
Toho University Omori Medical Center
Ōta-ku, Tokyo, Japan, 143-8541
Actively Recruiting
12
Kyoto University Hospital
Kyoto, Japan, 606-8507
Actively Recruiting
13
Hanyang University Seoul Hospital
Seoul, South Korea, 04763
Actively Recruiting
14
Seoul National University Hospital
Seoul, South Korea, 3080
Actively Recruiting
15
Kaohsiung Chang Gung Memorial Hospital
Kaohsiung City, Taiwan, 83301
Actively Recruiting
16
Taipei Veterans General Hospital
Taipei, Taiwan, 11211
Actively Recruiting
Research Team
C
Clinical Trials Administrator
CONTACT
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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