Plain-English translation of NCT06351592 on ClinicalTrials.gov β Β· Source last updated Β· Translation generated Β· How we translate trials
Phase 1/2 β 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.
This trial is testing , a new experimental treatment given as an injection into the fluid around your spinal cord, for people with a specific type of ALS caused by changes in the SOD1 gene. This is the first time this medication will be used in humans. The main goal is to see if the treatment is safe and well-tolerated, and to learn how it affects disease markers and symptoms.
SOD1-related ALS is a rare genetic form of this serious disease, and there are very few treatment options available. This medication is designed to target the root genetic cause, and researchers hope it may help slow or reduce symptoms.
You likely qualify ifβ¦
You likely don't qualify ifβ¦
You will receive injections directly into the fluid around your spinal cord (via lumbar puncture or spinal tap) as part of a 4-week treatment period. You will be randomly assigned to receive either the medication or a placebo (inactive treatment), and neither you nor the research team will know which one you receive. The study will involve multiple visits for safety checks, blood tests, spinal fluid sampling, and assessments of your ALS symptoms and disease markers.
AI-generated summary from trial data Β· Jun 1, 2026 Β· Not medical advice
Australia
Belgium
Canada