Actively Recruiting
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
Led by Praxis Precision Medicines · Updated on 2026-05-27
40
Participants Needed
10
Research Sites
130 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating the safety and effectiveness of elsunersen in children with early onset SCN2A Developmental and Epileptic Encephalopathy (DEE), a rare neurological condition characterized by frequent seizures starting before three months of age. This phase 3 clinical trial focuses on pediatric participants who have a specific genetic mutation called Gain of Function SCN2A variant confirmed by genetic testing. The study aims to see how elsunersen affects seizure frequency and other related measures over 24 weeks. Participants will receive open-label treatment with elsunersen administered intrathecally every four weeks for 24 weeks. Two dosing options are studied: 1 mg and 0.5 mg of elsunersen. The trial has multiple cohorts, each receiving the open-label treatment during the experimental period. There is no placebo group, and all participants will be monitored closely throughout the study. During the trial, participants will be evaluated on seizure frequency, clinical global impression scales, sleep assessments, and other efficacy measures at various time points over 24 weeks. Safety and tolerability of elsunersen will also be assessed throughout this period. The total participation duration covers the 24-week treatment and observation period, with ongoing evaluations to understand the drug's effects in this pediatric population.
CONDITIONS
Brief Title
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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.
You will not qualify if you...
- 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.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 24 weeks
Participants receive elsunersen treatment intrathecally every 4 weeks for 24 weeks.
6 visits every 4 weeks (in-person)
Trial Site Locations
Total: 10 locations
1
Praxis Research Site
San Diego, California, United States, 92123
Actively Recruiting
2
Praxis Research Site
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Praxis Research Site
Chicago, Illinois, United States, 60612
Actively Recruiting
4
Praxis Research Site
Cincinnati, Ohio, United States, 45229
Actively Recruiting
5
Praxis Research Site
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
6
Praxis Research Site
Memphis, Tennessee, United States, 38105
Actively Recruiting
7
Praxis Research Site
Porto Alegre, Brazil, 90035
Withdrawn
8
Praxis Research Site
São Paulo, Brazil, 05403
Withdrawn
9
Praxis Research Site
Bonn, Bonn, Germany, 53127
Actively Recruiting
10
Praxis Research Site
Rome, Italy, 00165
Actively Recruiting
Research Team
H
Head of Pharmacovigilance
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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