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A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety

NCT07019922

RecruitingPhase 3

Sponsor: Praxis Precision Medicines

Conditions: Epileptic Encephalopathy, SCN2A Encephalopathy

Interventions: 1mg elsunersen, 0.5mg elsunersen

Countries: United States, Brazil, Germany, Italy

A Multi-Center, Single-Arm Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy

Eligibility overview

Sex: ALL

Age: 1 Day to 18 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

Inclusion Criteria:

* Has a documented Gain of Function SCN2A variant confirmed through genetic testing.
* Has onset of seizures prior to 3 months of age.
* Seizure frequency of 4 or more countable motor seizures per 28-day during the Baseline Observation Period.

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.
* Has bone, spine (eg, kyphosis, scoliosis), bleeding, or other disorder.
* Has received any experimental or investigational drug, device, or other therapy within 30 days or 5 half-lives (whichever is longer) prior to Screening, including any prior use of gene therapy.
* Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial.

Locations (10)

San Diego, California, United States
Aurora, Colorado, United States
Chicago, Illinois, United States
Cincinnati, Ohio, United States
Philadelphia, Pennsylvania, United States
Memphis, Tennessee, United States
Porto Alegre, Brazil
São Paulo, Brazil
Bonn, Bonn, Germany
Rome, Italy