NCT05737784PHASE1, PHASE2Recruiting
A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
Praxis Precision Medicines
Start Date
4/13/2023
Completion Date
9/1/2027
Summary
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
Age Range: 2 years to 18 years
Inclusion Criteria:
* 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
Exclusion Criteria:
* 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
Interventions
DRUG
PRAX-222 - Initial Dose
DRUG
PRAX-222 - Initial Ascending Doses
DRUG
PRAX-222 - Optional Ascending Doses
DRUG
PRAX-222 - Fixed Doses
PROCEDURE
Placebo
Conditions
SCN2A-DEEEpilepsy
Locations
Le Bonheur Childrens Hospital
Memphis, Tennessee 38103
United States
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul 90035-003
Brazil
Praxis Research Site
São Paulo, 05403-010
Brazil