Testing PRAX-562 for pediatric epilepsy caused by SCN2A and SCN8A variants
Official title: A Phase 2,Double-Blind,Randomized Clinical Trial to Explore the Safety,Tolerability,Efficacy, and Pharmacokinetics of PRAX-562 in Pediatric Participants With Developmental and Epileptic Encephalopathies Followed by Open-Label Extension(OLE)
Run by Praxis Precision Medicines · NCT05818553 · Data updated 2026-05-14
This study is no longer in our active list. It may have stopped recruiting or finished. The details below are as of 2026-05-14 and are kept for reference. Check ClinicalTrials.gov for its current status, or see similar studies below.
This study is testing a new medication called PRAX-562 to see if it can help children with epilepsy caused by certain genetic changes feel better and have fewer seizures.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Ages | 2 Years to 18 Years |
| Sex | All |
| Enrollment | 100 (estimated) |
| Sponsor | Praxis Precision Medicines Industry-sponsored |
| Locations | 5 sites (Atlanta, Georgia and 4 other locations) |
| Trial ID | NCT05818553 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are pediatric patients with documented SCN2A or SCN8A variants and a history of early-onset seizures.
✕Probably not a fit
Patients with other significant genetic variants contributing to their epilepsy or developmental disorder may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This clinical trial evaluates the safety, tolerability, efficacy, and pharmacokinetics of PRAX-562 in children suffering from seizures associated with SCN2A and SCN8A developmental and epileptic encephalopathies. It is a Phase 2 and Phase 3, double-blind, randomized trial that includes participants with specific genetic variants and seizure frequency criteria. The study aims to provide insights into the potential benefits of PRAX-562 for managing these rare forms of epilepsy.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce seizure frequency and improve the quality of life for children with SCN2A and SCN8A encephalopathies.
How similar studies have gone: While this approach is focused on specific genetic variants, similar studies targeting genetic causes of epilepsy have shown promising results.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Has a documented rare missense variant in SCN2A with onset of seizures occurring in the first three months of life or has a documented de novo (not observed in either parent) missense variant in SCN8A with onset of seizures occurring in the first six months of life. * Has a seizure frequency as follows: * At least 8 countable motor seizures in the 4 weeks immediately prior to Screening as reported by the parent/legal guardian or in the opinion of the investigator AND * At least 8 countable motor seizures during the 28 day Baseline Observation Period (during which seizure frequency is recorded in a daily seizure diary). * Additional inclusion criteria apply and will be assessed by the study team. Exclusion Criteria: * Has any clinically significant or known pathogenic or likely pathogenic genetic variant other than in SCN2A and SCN8A or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder. * Has a documented, functionally characterized loss-of-function (LoF) missense variant or a presumed LoF variant (nonsense or frameshift variant) based on genetic testing and/or clinical evidence that prior exposure to a sodium channel blocker (SCB) medication worsened seizures. * Has 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening. * Additional exclusion criteria apply and will be assessed by the study team.
Where it's running
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Praxis Research Site
Chicago, Illinois, United States Recruiting -
Praxis Research Site
Minneapolis, Minnesota, United States Recruiting -
Praxis Research Site
Hackensack, New Jersey, United States Recruiting -
Praxis Research Site
Madrid, Spain Recruiting -
Praxis Research Site
Atlanta, Georgia, United States Active not recruiting
Study contacts
- Study coordinator: Head of Pharmacovigilance
- Email: clinicaltrials@praxismedicines.com
- Phone: 617-300-8460
Similar studies
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This trial tests whether elsunersen can reduce seizures and is safe in infants and young children with early-onset SCN2A-related developmental and epileptic encephalopathy who have gain-of-function variants.
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.