A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Recruiting · NCT07019922 · Interventional (participants receive a specific treatment) · Lead sponsor: Praxis Precision Medicines
View the official record on ClinicalTrials.gov →Interventions studied
0.5mg elsunersen1mg elsunersen
What this trial is about
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
Who can take part
Age range
1 Day to 18 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 3
Study type
Interventional (participants receive a specific treatment)
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.
Where it is running
10 locations listed across 6 US states.
- Praxis Research Site - San Diego, California, United States
- Praxis Research Site - Aurora, Colorado, United States
- Praxis Research Site - Chicago, Illinois, United States
- Praxis Research Site - Cincinnati, Ohio, United States
- Praxis Research Site - Philadelphia, Pennsylvania, United States
- Praxis Research Site - Memphis, Tennessee, United States
- Praxis Research Site - Porto Alegre, Brazil
- Praxis Research Site - Rome, Italy
- Praxis Research Site - São Paulo, Brazil
- Praxis Research Site - Bonn, Bonn, Germany