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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

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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