Plain-English translation of NCT07531745 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · 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 study is testing a new medication called that is delivered directly into the fluid around the spinal cord through a single injection. Researchers want to learn if this treatment is safe and how well it works in children ages 2 to 12 who have Dravet syndrome, a rare and severe form of childhood epilepsy. The study has two main phases: first, researchers will test increasing doses in smaller groups to find the right amount; then, qualified participants may continue with multiple doses over 2 years.
Dravet syndrome is a severe type of epilepsy that starts in infancy and causes frequent, difficult-to-control seizures. Many children do not respond well to standard seizure medications, so doctors are looking for new treatment options like this medication to help reduce seizures and improve quality of life.
You likely qualify if…
You likely don't qualify if…
In Part 1 (6 months), your child will receive a single spinal injection of the medication at one of four dose levels, with frequent monitoring visits to check safety and how the body responds. If all goes well, your child may be invited to continue in Part 2 (24 months), where they will receive multiple doses of the same medication spaced over time, followed by 7 months of additional safety follow-up. Throughout the study, your child will have regular clinic visits, blood tests, and seizure monitoring to ensure the treatment is working safely.
AI-generated summary from trial data · Jul 1, 2026 · Not medical advice
United States
Phase
Safety & dosing / Early efficacy
Sponsor
Ionis Pharmaceuticals, Inc.
Enrollment target
~32 participants
Started
May 2026
Primary completion
December 2030
Age range
2 Years – 12 Years
Last updated on clinicaltrials.gov in June 2026.
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Central contact
Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc.
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