A Clinical Trial to Investigate the Efficacy, Safety and Pharmacokinetics of Elsunersen in Children
Research type
Research Study
Full title
A Phase 3, 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
IRAS ID
1010595
Contact name
William Motel
Contact email
Sponsor organisation
Praxis Precision Medicines
Research summary
This study will investigate a new drug, called Elsunersen (formerly knows as PRAX-222), for the treatment of children with a rare and serious brain disease called SCN2A developmental and epileptic encephalopathy, which is caused by mutations in the gene SCN2A. There is currently no medicine approved for treatment of these patients, and Elsunersen has shown positive effects in earlier studies in patients in controlling epileptic fits (seizures). The developer of Elsunersen, Praxis Precision Medicines, is the sponsor of this study. The study will assess the effectiveness (efficacy), safety, how well the body can handle Elsunersen (tolerability) and examine what happens to Elsunersen in the human body after administration (pharmacokinetics). Elsunersen is a short sequence of nucleic acids, known as an antisense oligonucleotide, and is injected into the spinal column (spinal canal). This study will be conducted at 2 NHS sites in Englaand and Scotland, and will enroll up to 5 patients, between 0 to 18 years of age. Patients will receive Elsunersen over an initial period of 24 weeks (treatment period), and, for those who choose to do so, another 24 weeks (treatment extension period). There will be a follow-up period of 4 weeks after a patient has finished treatment. As well as receiving Elsunersen, during the study patients will undergo physical examinations and clinical measurements (for example electrocardiogram assessment), and samples of blood and cerebrospinal fluid will be taken from patients for safety and efficacy assessments. Patients, or their caregivers, will also need to keep a diary of seizures experienced throughout the study, and report any side effects (adverse events).
REC name
North West - Haydock Research Ethics Committee
REC reference
25/NW/0076
Date of REC Opinion
30 May 2025
REC opinion
Further Information Favourable Opinion