A Seamless, Clinical Trial to Investigate the Safety and Efficacy of Multiple Doses of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
Praxis Precision Medicines
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
- Age range
- 2–18 years
- Sex
- All
- Healthy volunteers
- No
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'…
Interventions
- DrugPRAX-222 - Initial Dose
PRAX-222
- DrugPRAX-222 - Initial Ascending Doses
Ascending doses of PRAX-222
- DrugPRAX-222 - Optional Ascending Doses
Escalation of PRAX-222 dose(s)
- DrugPRAX-222 - Fixed Doses
Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222
- ProcedurePlacebo
Placebo procedure
Locations (3)
- Le Bonheur Childrens HospitalMemphis, Tennessee
- Hospital de Clinicas de Porto AlegrePorto Alegre, Rio Grande do Sul
- Praxis Research SiteSão Paulo