RC001 for children with Dravet syndrome
Part of Brain & nervous system clinical trials.
This early-phase trial tests an experimental medicine called RC001 in children with Dravet syndrome caused by SCN1A gene changes. The goal is to see if RC001 is safe and how the body processes it, with the hope of reducing seizures.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child is aged 2 to 18 years and has Dravet syndrome caused by a specific SCN1A gene change.
- They have had at least 6 seizures in the past 12 weeks, and at least 2 in the last 4 weeks.
- They have tried at least one epilepsy treatment (medication, ketogenic diet, or vagus nerve stimulation) and it didn't work well enough or caused bad side effects.
- They are not taking certain seizure medicines that block sodium channels (like carbamazepine, lamotrigine, or rufinamide).
- They haven't had gene therapy, cell therapy, or any vaccine in the past few weeks.
- They do not have other serious uncontrolled health conditions or a history of certain brain or spinal problems.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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