A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age1-18
SponsorPraxis Precision Medicines
About this trial
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 criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- 1mg elsunersen
- 0.5mg elsunersen
Treatment groups
40 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov