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
This trial does not accept healthy volunteersQualifiers
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
Parallel
Treatments tested in this trial
PRAX-222 - Initial Dose
DrugPRAX-222
PRAX-222 - Initial Ascending Doses
DrugAscending doses of PRAX-222
PRAX-222 - Optional Ascending Doses
DrugEscalation of PRAX-222 dose(s)
PRAX-222 - Fixed Doses
DrugFixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222
Placebo
Procedure/SurgeryPlacebo procedure
Treatment groups
8
Treatment groupsSee each treatment group below.
Trial outcomes
Primary outcomes
Number of participants with treatment-emergent adverse events (Preliminary Safety, Dose Escalation)
The number of participants with treatment-emergent adverse events will be reported by severity and preferred term.
Seizure frequency (Confirmatory Phase)
Seizure frequency will be captured by a seizure diary and outcomes will be measured by summing the seizure frequency over a 28-day time period following the 6th dose administration in the confirmatory phase.
Secondary outcomes
Seizure frequency (Preliminary Safety)
Seizure frequency will be captured by a seizure diary and outcomes will be measured by summing the seizure frequency over a 28-day time period following the 4th dose administration in the preliminary safety phase.
Seizure frequency (Preliminary Safety)
Seizure frequency will be captured by a seizure diary and outcomes will be measured by summing the seizure frequency over a 28-day time period following each dose administration in the preliminary safety phase.
Percent change in seizure frequency (Preliminary Safety)
Seizure frequency will be captured by a seizure diary and outcomes will be measured by summing the seizure frequency over a 28-day time period following each dose administration in the preliminary safety phase. Percent change in seizure frequency will be calculated using a 4-week baseline observation period during Screening as the baseline measure.
Number of participants with a treatment response (Preliminary Safety)
Seizure frequency will be captured by a seizure diary and outcomes will be measured by summing the seizure frequency over a 28-day time period following each dose administration in the preliminary safety phase. Percent change in seizure frequency will be calculated using a 4-week baseline observation period during Screening as the baseline measure. Treatment response will be defined as a \>=50% reduction in seizure frequency.
Sponsors and contacts
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