Testing a new nerve-protecting medication for ALS in people without SOD1 mutations

Plain-English translation of NCT07294144 on ClinicalTrials.gov ↗ · Source last updated June 2026 · Translation generated June 2026 · How we translate trials

Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.

What is this trial?

This trial is testing whether a medication called can help people with ALS (amyotrophic lateral sclerosis) who don't have SOD1 mutations. is already approved to treat a specific type of ALS caused by SOD1 mutations, but doctors want to know if it could help a broader group of people with ALS. The study will measure whether the medication lowers a specific marker of nerve damage in the blood and spinal fluid.

Most ALS treatments are designed for specific genetic forms of the disease, but many people with ALS don't have these known genetic mutations. This trial exists to see if this medication, which works well for one genetic type of ALS, might also help people whose ALS isn't caused by the genes currently being treated.

Do you qualify?

You likely qualify if…

you are 18 years or older
you have been diagnosed with ALS within the past 24 months
you have had genetic testing showing you do not have SOD1 or FUS mutations
your lung function (measured by a breathing test) is at least 50% of normal
you are able to attend all study visits and follow the study schedule

You likely don't qualify if…

you are currently taking other experimental ALS treatments or have taken them in the past month
you are pregnant, breastfeeding, or unwilling to use contraception for 5 months after treatment ends
you have had recent drug or alcohol abuse (within 6 months) that would affect your ability to participate
you have a significant active infection requiring antibiotics or antivirals
you have serious cognitive problems, untreated depression, or recent suicidal thoughts

What participation looks like

You will receive 8 doses of the medication over 24 weeks, given through a lumbar puncture (spinal injection) at weeks 0, 2, 4, 8, 12, 16, 20, and 24. You'll complete follow-up visits at weeks 28 and 32 after your last dose. Throughout the study, you'll undergo various tests including strength and breathing assessments, blood draws, and questionnaires about how you're doing.

AI-generated summary from trial data · Jun 12, 2026 · Not medical advice

Testing a new nerve-protecting medication for ALS in people without SOD1 mutations

What is this trial?

Do you qualify?

What participation looks like

Trial locations(2 sites)