Testing PRAX-222 for treating early onset SCN2A epilepsy in children
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
PHASE1; PHASE2 · Praxis Precision Medicines · NCT05737784
This study is testing a new medication called PRAX-222 to see if it can help children aged 2 to 18 with early onset SCN2A epilepsy by reducing their seizures and improving their development.
Quick facts
| Phase | PHASE1; PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 2 Years to 18 Years |
| Sex | All |
| Sponsor | Praxis Precision Medicines (industry) |
| Locations | 3 sites (Memphis, Tennessee and 2 other locations) |
| Trial ID | NCT05737784 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety and efficacy of PRAX-222 in pediatric participants aged 2 to 18 years who have early onset SCN2A developmental and epileptic encephalopathy. The study will involve administering various doses of PRAX-222, including initial, ascending, and fixed doses, compared to a placebo. Participants must have a documented SCN2A variant and have experienced seizures before 3 months of age. The trial will assess the impact of the treatment on seizure frequency and developmental outcomes.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients aged 2 to 18 years with a documented SCN2A variant and early onset seizures.
Not a fit: Patients with significant genetic variants other than SCN2A or those on more than two sodium channel blocking anti-seizure medications may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce seizure frequency and improve developmental outcomes for children with SCN2A-DEE.
How similar studies have performed: Other studies involving antisense oligonucleotides for genetic epilepsy have shown promise, suggesting potential success for this approach.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Memphis, Tennessee and 2 other locations
- Le Bonheur Childrens Hospital — Memphis, Tennessee, United States (COMPLETED)
- Hospital de Clinicas de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil (RECRUITING)
- Praxis Research Site — São Paulo, Brazil (RECRUITING)
Study contacts
- Study coordinator: Head of Pharmacovigilance
- Email: clinicaltrials@praxismedicines.com
- Phone: 617-300-8460
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: SCN2A-DEE, Epilepsy, Antisense Oligonucleotide, NaV1.2 Voltage-Gated Sodium Channel, Voltage-Gated Sodium Channel Blockers, SCN2A variant, Pediatric epilepsy, Developmental and Epileptic Encephalopathy