Revolutionizing the standard of care for children with early-onset SCN2A Developmental and Epileptic Encephalopathy (DEE)
The EMBRAVE 3 study is currently enrolling to evaluate a new potential treatment targeting the underlying cause of disease in early-onset SCN2A DEE.
About the EMBRAVE 3 Study
Purpose
To understand how safe and effective elsunersen is in reducing seizures and improving other symptoms associated with SCN2A gain-of-function DEE
Duration
Up to 24 weeks in the initial study, with the opportunity to continue to stay on treatment for an additional 24 weeks
In Clinic
United States, South America, Europe, United Kingdom
Relocation options are available for families that reside outside of the above geographies
About Elsunersen
Elsunersen is an investigational antisense oligonucleotide (ASO) with the potential to be the first disease-modifying treatment for early-onset gain-of-function SCN2A DEE, designed to selectively decrease SCN2A gene expression. By targeting the underlying genetic cause of disease, elsunersen has demonstrated potential to go beyond seizures to treat other symptoms of the disease.
About ASOs
ASOs are short, lab-made strands of genetic material that can target specific genes and adjust how they behave. They are custom-designed to match the genetic error causing the disease. That means they don’t just treat symptoms, they go after the root cause. Elsunersen, a type of ASO, is designed to reduce the levels of SCN2A gene expression in children with SCN2A gain-of-function mutations. By lowering how much of the SCN2A protein is made, elsunersen helps quiet the excessive electrical activity in the brain, which can reduce the seizure burden and improve quality of life with SCN2A gain-of-function mutations.
EMBRAVE 3 Study Criteria
-
Are 0 through 18 years old - Have received a diagnosis of a SCN2A gene mutation with onset of seizures in the first 3 months of life
- Have at least 4 motor seizures (seizures that involve movement) in the 4 weeks prior to screening
Why should my child participate?
- The study is designed to reduce the burden of participation by offering a combination of in-clinic visits and at-home telehealth visits
- Travel and/or relocation assistance available. This will include lodging, meals, and any other costs associated with study participation will be paid for by the sponsor
- Option to participate in a 24-week open-label extension, in which all participants will receive elsunersen (study drug)
- Opportunity to potentially change the future for children diagnosed with SCN2A DEE
