Testing PRAX-562 for pediatric epilepsy caused by SCN2A and SCN8A variants

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)

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
• Enrollment: 100 (estimated)
• Ages: 2 Years to 18 Years
• Sex: All
• Sponsor: Praxis Precision Medicines (industry)
• Locations: 5 sites (Atlanta, Georgia and 4 other locations)
• Trial ID: NCT05818553 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients with documented SCN2A or SCN8A variants and a history of early-onset seizures.

Not a fit: Patients with other significant genetic variants contributing to their epilepsy or developmental disorder may not benefit from this study.

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 performed: While this approach is focused on specific genetic variants, similar studies targeting genetic causes of epilepsy have shown promising results.

Eligibility 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 this trial is running

Atlanta, Georgia and 4 other locations

• Praxis Research Site — Atlanta, Georgia, United States (ACTIVE_NOT_RECRUITING)
• 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)

Study contacts

• Study coordinator: Head of Pharmacovigilance

Conditions: SCN2A Encephalopathy, SCN8A Encephalopathy, Pediatric epilepsy, NaV1.2 Voltage-Gated Sodium Channel, SCN2A variant, SCN8A variant, Developmental and epileptic encephalopathy