Plain-English translation of NCT07401121 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This is one of the first times this treatment has been tested in people.
This trial is testing CTx1000, an experimental gene therapy designed for people with ALS (a disease that affects nerve cells controlling movement). The treatment is delivered as a single injection directly into the fluid surrounding the spinal cord. Researchers want to see if this medication is safe and how well patients tolerate it.
ALS currently has no cure, and available treatments offer only modest benefits. This trial exists to explore whether targeting and breaking down a specific protein called TDP-43 — which accumulates in ALS — could help slow or stop the disease.
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If you join this trial, you will receive one injection of the experimental medication delivered directly into the space around your spinal cord. The study is open-label, meaning you and your doctors will know you are receiving the treatment. You will be closely monitored to assess safety and how your body responds to the medication, though the trial record does not specify the exact number or timing of follow-up visits.
AI-generated summary from trial data · Jun 10, 2026 · Not medical advice
Australia
Phase
Safety & dosing
Sponsor
Celosia Therapeutics Pty Ltd
Collaborators
Novotech (Australia) Pty Limited
Enrollment target
~15 participants
Started
January 2026
Primary completion
December 2027
Age range
18 Years – 80 Years
Last updated on clinicaltrials.gov in February 2026.
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