Testing a new gene therapy injection for inherited nerve disease

Plain-English translation of NCT06351592 on ClinicalTrials.gov ↗ · Source last updated September 2026 · Translation generated August 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 study in humans testing a new medication called , delivered as an injection into the fluid surrounding your spinal cord. The treatment is designed for people with a specific type of ALS caused by changes in the SOD1 gene. Researchers want to see if this medication is safe, how your body handles it, and whether it might help slow the progression of your symptoms.

Currently, there are very few treatment options for people with SOD1-related ALS, and this medication represents a new approach specifically designed to target the genetic cause of this form of the disease. By testing this treatment early, researchers hope to find a way to help people with this genetic form of ALS before it progresses further.

Do you qualify?

You likely qualify if…

you have been diagnosed with ALS and carry a SOD1 gene mutation known to cause the disease
your lung function (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 normal blood pressure readings
your platelet count is above 50,000 per microliter of blood

You likely don't qualify if…

you have had a tracheostomy (breathing tube inserted in your windpipe)
you have dementia or uncontrolled mental health conditions such as active suicidal thoughts or untreated depression
you have had brain or spinal cord injury or disease that would make spinal fluid collection unsafe
you have had an implanted shunt or catheter in your brain or spinal cord
you received a different SOD1 ALS treatment (tofersen) within the past 6 months

What participation looks like

You would receive spinal injections of either the study medication or a placebo (inactive substance) during a 4-week treatment period, with researchers gradually testing higher doses in different groups. You'll have blood tests and spinal fluid samples collected to measure how the medication works in your body and check for any effects on disease markers. Visits will include safety checks, symptom assessments, and monitoring for any side effects throughout the study period.

AI-generated summary from trial data · Aug 7, 2026 · Not medical advice

Testing a new gene therapy injection for inherited nerve disease

What is this trial?

Do you qualify?

What participation looks like

Trial locations(22 sites)