New drug trial aims to tame seizures in rare childhood disorder
NCT ID NCT05249556
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether the drug ganaxolone can reduce seizures in children aged 6 months to 2 years with CDKL5 deficiency disorder, a rare genetic condition causing hard-to-control seizures. Twenty children will receive either ganaxolone or a placebo alongside their usual seizure medication for 12 weeks. Researchers will measure changes in seizure frequency and monitor safety.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ganaxolone
What this could lead to
If it works, this could provide a new option to reduce seizures in very young children with CDKL5 deficiency disorder.
What could go wrong
This is a small early-stage trial with only 20 participants, so results may not apply to all children. The drug may not work better than placebo or could cause side effects.
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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