Testing a new spinal fluid treatment for ALS in adults

Plain-English translation of NCT07855211 on ClinicalTrials.gov ↗ · Source last updated October 2026 · Translation generated October 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.

What is this trial?

This study is testing a new medication called that is injected directly into the spinal fluid to treat adults with ALS (amyotrophic lateral sclerosis). The trial will first test different doses of the medication to see if it is safe and well-tolerated, and then follow participants for a long time to see if the treatment helps slow disease progression.

ALS is a serious disease that causes nerve cells to break down and stop working over time. There are very few treatment options available, so researchers are testing this medication to see if it can help protect nerve cells and slow the decline that people with ALS experience.

Do you qualify?

You likely qualify if…

you have been diagnosed with ALS according to established medical criteria
you have at least 50% of the expected lung function for your age, sex, and height
you do not carry specific genetic mutations in the SOD1 or FUS genes
you are not on a ventilator for more than 22 hours per day
you have not received other experimental ALS treatments within the past month

You likely don't qualify if…

you have HIV or a history of blood clots in your legs or lungs within the past 2 years
you have another progressive nerve or muscle disease besides ALS
you need or are planning to get a diaphragm pacing device during the study
you are currently taking other experimental ALS drugs or treatments
you have serious abnormalities in blood tests or heart function

What participation looks like

You would receive injections of either the new medication or a placebo directly into your spinal fluid over 12 to 20 weeks, depending on which group you're assigned to. After this initial period, you would be invited to continue receiving the medication for up to several years, with regular visits to the clinic for safety checks, blood tests, and measurements of how your disease is progressing. The study tracks both how well you tolerate the treatment and whether it affects the course of your ALS.

AI-generated summary from trial data · Oct 22, 2026 · Not medical advice

Trial locations(2 sites)

United States

Washington University School of Medicine_St. Louis, St Louis, Missouri

Austin Neuromuscular Center_Austin, Austin, Texas

View full record on ClinicalTrials.gov