Gene therapy hopes to tame severe childhood epilepsy

NCT ID NCT07675746

Recruiting now Disease control Sponsor: Second Affiliated Hospital of Guangzhou Medical University Source: ClinicalTrials.gov ↗

First seen Jun 30, 2026 · Last updated Jul 01, 2026 · Updated 1 time

Summary

This early-stage trial tests a gene therapy called RC001 in children aged 2 to 18 with Dravet syndrome, a severe form of epilepsy caused by a genetic mutation. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and how the body processes the drug, with a small group of 8 participants receiving increasing or fixed doses.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: RC001 (gene therapy)
What this could lead to: If successful, this could point toward a treatment that reduces seizures in children with Dravet syndrome.
What could go wrong: This is a very early, small trial (8 people) focused on safety. It may not show clear benefit, and gene therapies carry risks like immune reactions or side effects from the injection.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Dravet syndrome Epilepsies, Myoclonic generalized epilepsy with febrile seizures plus, type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DRAVET SYNDROME (DS)

Contacts and locations

Locations

The Second Affiliated Hospital of Guangzhou Medical University
RECRUITING

Guangzhou, Guangdong, 510120, China

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