RECRUITINGPhase 1 / Phase 2INTERVENTIONAL
A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
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
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.
Who May Be Eligible (Plain English)
Who May Qualify:
- 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
Who Should NOT Join This Trial:
- 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
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
DRUG
PRAX-222 - Initial Dose
PRAX-222
DRUG
PRAX-222 - Initial Ascending Doses
Ascending doses of PRAX-222
DRUG
PRAX-222 - Optional Ascending Doses
Escalation of PRAX-222 dose(s)
DRUG
PRAX-222 - Fixed Doses
Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222
PROCEDURE
Placebo
Placebo procedure
Locations (3)
Le Bonheur Childrens Hospital
Memphis, Tennessee, United States
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil
Praxis Research Site
São Paulo, Brazil