RecruitingPhase 1Phase 2NCT05737784

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

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


Sponsor

Praxis Precision Medicines

Enrollment

60 participants

Start Date

Apr 13, 2023

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 2 YearsMax Age: 18 Years

Inclusion Criteria3

  • 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.

Exclusion Criteria2

  • 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

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Interventions

DRUGPRAX-222 - Initial Dose

PRAX-222

DRUGPRAX-222 - Initial Ascending Doses

Ascending doses of PRAX-222

DRUGPRAX-222 - Optional Ascending Doses

Escalation of PRAX-222 dose(s)

DRUGPRAX-222 - Fixed Doses

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

PROCEDUREPlacebo

Placebo procedure


Locations(3)

Le Bonheur Childrens Hospital

Memphis, Tennessee, United States

Hospital de Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, Brazil

Praxis Research Site

São Paulo, Brazil

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NCT05737784


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