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Clinical Trials in the USA / NCT05737784
Active, not recruiting Phase 1/2

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

NCT05737784 · tracked via the Priya Life Science USA tracker
Sponsor
Praxis Precision Medicines
Phase
Phase 1/2
Started
2023-04-13
Last updated
2026-08-03

Condition(s) studied

SCN2A-DEEEpilepsy

Investigational drug(s) / intervention(s)

PRAX-222 - Initial DosePRAX-222 - Initial Ascending DosesPRAX-222 - Optional Ascending DosesPRAX-222 - Fixed DosesPlacebo

PRAX-222 - Initial Dose: PRAX-222

PRAX-222 - Initial Ascending Doses: Ascending doses of PRAX-222

PRAX-222 - Optional Ascending Doses: Escalation of PRAX-222 dose(s)

PRAX-222 - Fixed Doses: Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222

Placebo: Placebo procedure

Study summary

The goal of this trial is to learn about the effect of PRAX-222 in pediatric participants with early onset SCN2A developmental and epileptic encephalopathy (DEE), aged 2 to 18 years.

Eligibility

Sex
ALL
Min age
2 Years
Max age
18 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • Number of participants with treatment-emergent adverse events (Preliminary Safety, Dose Escalation) — Screening (-8 weeks) through up to 92 weeks
    The number of participants with treatment-emergent adverse events will be reported by severity and preferred term.
  • Seizure frequency (Confirmatory Phase) — 36 to 40 weeks
    Seizure frequency will be captured by a seizure diary and outcomes will be measured by summing the seizure frequency over a 28-day time period following the 6th dose administration in the confirmatory phase.

Trial sites (3)

FacilityCityRegionStatus
Le Bonheur Childrens Hospital Memphis Tennessee
Hospital de Clinicas de Porto Alegre Porto Alegre Rio Grande do Sul
Praxis Research Site São Paulo Brazil

More Praxis Precision Medicines trials in the USA

A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE) Active, not recruiting
Randomized Clinical Trial Evaluating the Efficacy and Safety of Vormatrigine in Adults With Focal Seizures Active, not recruiting
Essential 3 - Decentralized, Phase 3 Study Evaluating the Safety and Efficacy of Ulixacaltamide in Essential Tremor (ET) Recruiting
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety Recruiting
Clinical Trial of Vormatrigine in Adult Patients With Epilepsy Enrolling by invitation

Other trials for the same condition

Diagnosing Epilepsy To EffeCT Change Long-Term Follow-Up Recruiting
Safety and Tolerability Study of SPN-817 in Adult Patients With Treatment Resistant Epilepsy Recruiting
Diagnosing Epilepsy To EffeCT Change Recruiting
Epilepsy Seizure Detection With Innovative Tripolar EEG (tEEG) Recruiting
A Safety, Tolerability, and Preliminary Efficacy of Low-intensity Focused Ultrasound Neuromodulation in Patients With Drug-resistant Epilepsy Recruiting
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 NCT05737784 on ClinicalTrials.gov ↗ ← All trials in the USA