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Clinical Trials in Australia / NCT06351592
Recruiting Phase 1/2

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 · tracked via the Priya Life Science Australia tracker
Phase
Phase 1/2
Started
2024-08-28
Last updated
2026-09-11

Condition(s) studied

Amyotrophic Lateral Sclerosis (ALS)Mutation in the Superoxide Dismutase-1 (SOD1) Gene

Investigational drug(s) / intervention(s)

ALN-SODDiluentPlacebo (PB)

ALN-SOD: Administered per the protocol

Diluent: Administered per the protocol

Placebo (PB): Administered per the protocol

Study summary

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

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (22)

FacilityCityRegionStatus
Concord Repatriation General Hospital Concord New South Wales Recruiting
Macquarie University Sydney New South Wales Recruiting
Sunshine Coast University Hospital Birtinya Queensland Recruiting
KU Leuven Leuven Vlaams-Brabant Recruiting
University of Alberta Hospital, Edmonton, Division of Neurology Edmonton Alberta Recruiting
University Hospital - London Health Sciences Centre London Ontario Recruiting
Sunnybrook Research Institute Toronto Ontario Recruiting
Montreal Neurological Institute and Hospital Montreal Quebec Recruiting
Universitaetsklinikum Ulm AoR Ulm Baden-Wurttemberg Recruiting
Hokkaido University Hospital Sapporo Hokkaido Recruiting
Tokushima University Hospital Tokushima Tokushima Recruiting
Toho University Omori Medical Center Ōta-ku Tokyo Recruiting
Kyoto University Hospital Kyoto Japan Recruiting
Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Neurologii Krakow Lesser Poland Voivodeship Recruiting
Medical University of Silesia Zabrze Silesian Voivodeship Recruiting
MTZ Clinical Research powered by Pratia Warsaw Poland Recruiting
Hanyang University Seoul Hospital Seoul South Korea Recruiting
Seoul National University Hospital Seoul South Korea Recruiting
Noorlands University Hospital-Department of Neurology Umeå Noorland Recruiting
Karolinska University Hospital, Karolinska Institutet-ALS Center-Department of Clinical Neuroscience Huddinge Stockholm County Recruiting
Kaohsiung Chang Gung Memorial Hospital Kaohsiung City Taiwan Recruiting
Taipei Veterans General Hospital Taipei Taiwan Recruiting

More Regeneron Pharmaceuticals trials in Australia

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06351592 on ClinicalTrials.gov ↗ ← All trials in Australia