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)
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
🩺 Plain-English Summary (8th-Grade Level)
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
Who can join: Review the eligibility criteria below.
Location: Concord Repatriation General Hospital — Concord, New South Wales
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (20 Sites)
- Concord Repatriation General Hospital — Concord, New South Wales
- Macquarie University — Sydney, New South Wales
- Sunshine Coast University Hospital — Birtinya, Queensland
- KU Leuven — Leuven, Vlaams-Brabant
- University of Alberta Hospital, Edmonton, Division of Neurology — Edmonton, Alberta
- University Hospital - London Health Sciences Centre — London, Ontario
- Sunnybrook Research Institute — Toronto, Ontario
- Montreal Neurological Institute and Hospital — Montreal, Quebec
- Universitaetsklinikum Ulm AoR — Ulm, Baden-Wurttemberg
- Hokkaido University Hospital — Sapporo, Hokkaido
- Tokushima University Hospital — Tokushima, Tokushima
- Toho University Omori Medical Center — Ōta-ku, Tokyo
- Kyoto University Hospital — Kyoto,
- Medical University of Silesia — Zabrze, Silesian Voivodeship
- MTZ Clinical Research powered by Pratia — Warsaw,
- Hanyang University Seoul Hospital — Seoul,
- Seoul National University Hospital — Seoul,
- Noorlands University Hospital-Department of Neurology — Umeå, Noorland
- Kaohsiung Chang Gung Memorial Hospital — Kaohsiung City,
- Taipei Veterans General Hospital — Taipei,
📞 Contact the Study Coordinator
Phone: (844) 734-6643
Email: clinicaltrials@regeneron.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Amyotrophic Lateral Sclerosis (ALS)
- HEALEY ALS Platform Trial - Regimen I NUZ-001 — Boston, Massachusetts
- A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS) — La Jolla, California
- Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY) — Phoenix, Arizona
- Resiliency Intervention for Patients With ALS and Their Care-Partners — Boston, Massachusetts
- Safety Study of XT-150 in Participants With ALS — Phoenix, Arizona
- A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS — Boston, Massachusetts
Explore by condition: All Amyotrophic Lateral Sclerosis (ALS) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.