About this trial
The goal of this trial is to learn about the effect of PRAX-222 in pediatric participants with early onset SCN2A developmental and epileptic encephalopathy (DEE), aged 2 to 18 years.
Eligibility criteria
Qualifiers
Has onset of seizures prior to 3 months of age.
Has a minimum weight of at least 10 kg at screening.
Has a documented SCN2A variant through genetic testing obtained via a laboratory accredited per Clinical Laboratory Improvement Amendments (CLIA) or College of American Pathologists (CAP) or equivalent.
Additional inclusion criteria apply and will be assessed by the study team
Disqualifiers
Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder.
Is taking more than 2 sodium channel blocking anti-seizure medications
Additional exclusion criteria apply and will be assessed by the study team
Trial design
Treatments tested in this trial
- PRAX-222 - Initial Dose
- PRAX-222 - Initial Ascending Doses
- PRAX-222 - Optional Ascending Doses
- PRAX-222 - Fixed Doses
- Placebo
Treatment groups
8
Treatment groupsSee each treatment group below.