Recruiting

Testing a new spinal injection treatment for severe childhood epilepsy

Plain-English translation of NCT07675746 on ClinicalTrials.gov ↗ · Source last updated June 2026 · Translation generated July 2026 · 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.

What is this trial?

This study is testing a new treatment called RC001 that is injected directly into the spinal fluid to help control seizures in children with Dravet syndrome, a severe form of epilepsy that starts in infancy. Researchers want to learn whether the medication is safe, how the body processes it, and whether it can reduce how often seizures happen. The study includes only 8 children and is one of the first steps in testing this approach.

Dravet syndrome is a rare and serious type of epilepsy that is very difficult to treat with standard anti-seizure medications alone, and many children continue to have frequent, life-threatening seizures despite current treatments. This trial explores a completely new approach—delivering medication directly to the spinal fluid—to see if it might help children whose seizures are not controlled by existing options.

Do you qualify?

You likely qualify if…

you are between 2 and 18 years old and have been diagnosed with Dravet syndrome caused by an SCN1A gene mutation
your seizures started before age 1 and include focal seizures, full-body shaking seizures, or other types of convulsions
you have had at least 6 seizures in the past 12 weeks and at least 2 seizures in the past 4 weeks
you have already tried at least one anti-seizure medication, special diet (like a ketogenic diet), or nerve stimulation therapy without good results
you are currently taking at least one anti-seizure medication at a stable dose and your other epilepsy treatments have not changed in at least 4 weeks

You likely don't qualify if…

you have certain genetic mutations in the SCN1A gene that work differently than the ones Dravet syndrome typically causes
you are currently taking sodium channel-blocking anti-seizure drugs like carbamazepine, oxcarbazepine, or lamotrigine
you have had gene therapy or cell therapy in the past year
you have received a vaccine in the past 12 weeks
you have liver problems or blood test results that show your liver is not working well

What participation looks like

You and your child would visit the research hospital for spinal injections of the medication. The study has two parts: first, a small group of 3 children receive gradually increasing doses to check for safety; then, a group of 5 children receive a set dose multiple times. Your child's seizures, safety, and how the body handles the medication will be closely monitored through visits and tests. After the main study ends, your child may have the option to continue receiving the medication if it seems to be helping and is safe.

AI-generated summary from trial data · Jul 13, 2026 · Not medical advice

Trial locations(1 site)

China

The Second Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong