Clinical trial NCT07019922: A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Official title: A Randomized, Multi-Center, Double-Blind, Sham-Procedure-Controlled Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Epileptic Encephalopathy
SCN2A Encephalopathy
Keywords: epilepsis
early onset scn2a
epileptic encephalopathy
scn2a
gain of function
seizure
Lead sponsor: Praxis Precision Medicines
Sponsor class: INDUSTRY
First posted: 2025-06-13
Enrollment: 50
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Specific milestones have to be met in Cohort 1 for enrollment to open in Cohort 2 and the same will apply to open Cohort 3.
Primary purpose: TREATMENT

Brief summary: A Randomized, Multi-Center, Double-Blind, Sham-Procedure-Controlled Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy


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.

Primary outcomes:
measure: To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE
timeFrame: 24 weeks
Secondary outcomes:
measure: To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE
timeFrame: 24 weeks
measure: To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE
timeFrame: 24 weeks
measure: CGI-S change from baseline
timeFrame: 24 weeks
measure: CGI subdomain scores at each postdose time point
timeFrame: 24 weeks
measure: CgGI-S from baseline
timeFrame: 24 weeks
measure: CgGI-I subdomain scores at each postdose time point
timeFrame: 24 weeks
measure: Sleep assessment scores from baseline
timeFrame: 24 weeks
measure: To evaluate the safety and tolerability of elsunersen in participants with early-onset SCN2A DEE
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Praxis Research Site
state: Illinois
Study officials:
affiliation: Praxis Precision Medicines
name: Medical Director
role: STUDY_DIRECTOR
References: