First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)

This is the first-ever human study of a new investigational drug called ALN-SOD — a gene-silencing therapy that reduces production of a malfunctioning protein (SOD1) — for people with ALS (amyotrophic lateral sclerosis, also known as Lou Gehrig's disease) caused by a mutation in the SOD1 gene. **You may be eligible if...** - You have ALS with a confirmed SOD1 gene mutation known or likely to cause the disease - Your lung breathing capacity is at least 50% of normal - Your BMI is 35 or below - If you are taking riluzole or edaravone, your dose has been stable for at least 4 weeks - Your blood counts and blood pressure are within normal ranges **You may NOT be eligible if...** - You are currently enrolled in another interventional clinical trial - You have had a tracheostomy (a breathing tube surgically placed in the throat) - You have severe kidney or liver disease - You have had a recent serious infection - You are pregnant or breastfeeding Talk to your doctor to see if this trial is right for you.