NCT07019922RecruitingPhase 3
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
Praxis Precision Medicines11 sites in 4 countries40 target enrollmentStarted: August 13, 2025Last updated:
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Praxis Precision Medicines
- Enrollment
- 40
- Locations
- 11
- Primary Endpoint
- To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE
Study Overview
Brief 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
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
Cohort 1 is the only masked cohort (40 participants). Cohort 2 and Cohort 3 are both open-label and will have 5 participants each.
Eligibility Criteria
- Ages
- 1 Day to 18 Years (Child, Adult)
- Sex
- All
- Accepts 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.
Arms & Interventions
Cohort 1 Arm 1: Open-Label Treatment Period
Experimental
Open-label elsunersen
Intervention: 1mg elsunersen (Drug)
Cohort 3: Open-Label Treatment Period
Experimental
Open-label elsunersen
Intervention: 0.5mg elsunersen (Drug)
Cohort 2: Open-Label Treatment Period
Experimental
Open-label elsunersen
Intervention: 1mg elsunersen (Drug)
Outcomes
Primary Outcomes
To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE
Time Frame: 24 weeks
Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks
Secondary Outcomes
- To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE(24 weeks)
- Sleep assessment scores from baseline(24 weeks)
- CgGI-I subdomain scores at each postdose time point(24 weeks)
- CGI-S change from baseline(24 weeks)
- CGI subdomain scores at each postdose time point(24 weeks)
- CgGI-S from baseline(24 weeks)
- To evaluate the safety and tolerability of elsunersen in participants with early-onset SCN2A DEE(24 weeks)
Investigators
Study Sites (11)
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