Testing a new treatment for a specific type of nerve disease caused by gene changes

Plain-English translation of NCT06351592 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated June 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

This is one of the first times this treatment has been tested in people.

What is this trial?

This is the first time is being given to people. The trial is testing whether this new medication is safe and well-tolerated in adults with SOD1-ALS, a form of ALS caused by a change in a specific gene. Researchers will also look at how the medication works in the body and whether it affects disease-related markers in blood and spinal fluid.

SOD1-ALS is a rare genetic form of a serious nerve disease that causes progressive muscle weakness. Currently, there are very few treatment options specifically designed for this genetic type, so this trial is testing whether this medication could offer a new approach to helping people with this condition.

Do you qualify?

You likely qualify if…

you have been diagnosed with ALS caused by a change in the SOD1 gene
you have weakness attributable to ALS
your lung function (measured as Slow Vital Capacity) is at least 50% of what is expected for your age, gender, and height
your body mass index is 35 or lower
you have a normal platelet count and normal blood pressure readings

You likely don't qualify if…

you have had a tracheostomy (breathing tube surgery)
you have dementia or uncontrolled psychiatric conditions including recent thoughts of suicide
you have a history of brain or spinal cord disease that would interfere with a spinal tap procedure
you have an implanted shunt or catheter in your brain or spinal cord
you have received a different ALS treatment called tofersen within the past 6 months

What participation looks like

Participants will receive injections of either the medication or a placebo directly into the spinal fluid through a procedure called a lumbar puncture. The study is divided into phases: first, researchers test different dose levels in small groups to find a safe dose, then expand to test the selected dose in more participants. Some participants will know they're receiving the medication (open-label), while others will be blinded to whether they receive the active drug or placebo for a 4-week treatment period. Throughout the study, participants will have blood tests and spinal fluid samples taken to measure how the body responds.

AI-generated summary from trial data · Jun 29, 2026 · Not medical advice

Testing a new treatment for a specific type of nerve disease caused by gene changes

What is this trial?

Do you qualify?

What participation looks like

Trial locations(20 sites)