Hope for infants: new drug trial targets devastating seizures in rare genetic disorder
NCT ID NCT05249556
Summary
This study is testing whether adding a new medicine called ganaxolone to a child's current seizure medications can better control seizures in infants with a rare genetic disorder called CDKL5 deficiency. It will involve about 20 infants aged 6 months to under 2 years old. For 12 weeks, some infants will receive ganaxolone while others receive a placebo (inactive substance), and researchers will carefully track changes in seizure frequency and safety.
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Conditions
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