Gene therapy hopes to tame severe childhood epilepsy
NCT ID NCT07675746
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.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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The Second Affiliated Hospital of Guangzhou Medical University
RECRUITINGGuangzhou, Guangdong, 510120, China
Contact Phone: •••-•••-•••• Email: •••••@•••••