Testing elsunersen in children with SCN2A-related epileptic encephalopathy
Tests elsunersen in young children with early onset SCN2A-related epilepsy and brain development problems.
Plain-language title generated from the registry record
Registry title: A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Key facts
- Where
- Porto Alegre and São Paulo, BrazilSee the 2 sites
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for children and teenagers with early onset SCN2A developmental and epileptic encephalopathy and a confirmed gain of function SCN2A variant.
- Age
- From 1 Day to 18 Years.
- Sex
- All sexes can join.
- Disease stage
- Seizures started before 3 months of age. At least 4 countable motor seizures per 28 days during the baseline observation period.
- Earlier treatment
- Cannot have taken any experimental or investigational drug, device or therapy within 30 days or 5 half-lives (whichever is longer) before screening. This includes any earlier gene therapy.
- Biomarker or test result
- A documented gain of function SCN2A variant confirmed by genetic testing.
Main reasons for not taking part
- Another clinically significant or known pathogenic genetic variant outside SCN2A
- A genetic variant that may explain or add to the epilepsy or developmental disorder
- Bone, spine (for example kyphosis, scoliosis), bleeding or other disorder
- Experimental drug, device or therapy within 30 days or 5 half-lives, including earlier gene therapy
- Pregnancy, breastfeeding or planning pregnancy during the trial
Read by machine on 9 November 2026.
Where in Brazil
- Porto AlegrePorto Alegre, BrazilNo longer taking part
- São PauloSão Paulo, BrazilNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Praxis Precision Medicines
- Trial code
- NCT07019922
- Record updated
- 3 August 2026
- Original description
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 EncephalopathyRead full original descriptionShow less
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
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
Inclusion: 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: 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; 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; Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial.