1mg elsunersen: 24 weeks every 4 weeks intrathecally
0.5mg elsunersen: 24 weeks every 4 weeks intrathecally
Study summary
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
Sex
ALL
Min age
1 Day
Max age
18 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE — 24 weeks Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks
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.
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