Ireland
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Active, not recruiting Phase 2/3

A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)

NCT05818553 · tracked via the Priya Life Science Spain tracker
Phase
Phase 2/3
Started
2023-08-02
Last updated
2026-01-29

Condition(s) studied

SCN2A EncephalopathySCN8A Encephalopathy

Investigational drug(s) / intervention(s)

PRAX-562 →

PRAX-562: Once daily oral or G-tube treatment.

Study summary

A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)

Eligibility

Sex
ALL
Min age
1 Year
Max age
18 Years
Healthy volunteers
No
Inclusion Criteria: * Has a documented variant in SCN2A with onset of seizures occurring in the first 3 months of life or has a diagnosis of SCN8A-DEE supported by both clinical and genetic findings. * 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 as documented in medical notes. AND o 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.

Primary outcome measure(s)

  • PART A (Cohorts 1 and 2) RDB: To evaluate the safety and tolerability of PRAX 562 in pediatric participants with SCN2A- and SCN8A- DEEs — 16 weeks
    Changes from baseline in monthly (28-day) motor seizure frequency
  • PART B (Cohorts 1 and 2) OLE: To evaluate the long-term safety and tolerability of PRAX-562 in pediatric participants with DEEs — 48 weeks
    Incidence and severity of TEAEs

Trial sites (8)

FacilityCityRegionStatus
Praxis Research Site Atlanta Georgia
Praxis Research Site Chicago Illinois
Praxis Research Site Minneapolis Minnesota
Praxis Research Site Hackensack New Jersey
Praxis Research Site Tel Litwinsky Israel
Praxis Research Site Madrid Spain
Praxis Research Site Glasgow United Kingdom
Praxis Research Site London United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05818553 on ClinicalTrials.gov ↗ ← All trials in Spain