A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy

Launched by PRAXIS PRECISION MEDICINES · Feb 10, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called PRAX-222 for children and teenagers aged 2 to 18 who have a specific type of epilepsy known as SCN2A developmental and epileptic encephalopathy (DEE). The main goal is to understand how PRAX-222 affects these young participants, especially since they have started having seizures before they turned 3 months old. The trial is currently looking for participants, and to qualify, children must weigh at least 10 kg and have a confirmed genetic change in the SCN2A gene, which is linked to their condition.

If your child is eligible and chooses to participate, they will receive the study drug and be monitored closely by the research team. There are some important things to note: children who have significant genetic issues beyond the SCN2A gene or are using more than two specific medications for seizures may not be able to join the study. This trial offers a chance for families to contribute to research that could help improve treatments for epilepsy in children.

Gender

ALL

Eligibility criteria

• 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