New hope for toddlers with rare epilepsy: ganaxolone trial begins
NCT ID NCT05249556
First seen Nov 21, 2025 · Last updated May 16, 2026 · Updated 19 times
Summary
This study tests whether adding ganaxolone to standard seizure medication can reduce seizures in children aged 6 months to 2 years with CDKL5 deficiency disorder, a rare genetic condition. About 20 children will receive either ganaxolone or a placebo for 12 weeks to see if it safely lowers seizure frequency. After the main phase, all children can receive ganaxolone in an open-label extension.
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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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