The EMBOLD Study
Hope for children with early-onset SCN2A and SCN8A Developmental and Epileptic Encephalopathies (DEE)
We understand the challenges you face in caring for a child with a rare DEE. The next phase of the EMBOLD study is enrolling early to investigate a potential new treatment for children with early-onset SCN2A and SCN8A DEEs.
Connect with a Nurse Navigator to schedule an appointment to determine your child's eligibility.
Topline data from the first part of EMBOLD study in SCN2A and SCN8A DEE
Children taking the study medication had nearly half as many seizures compared with those not taking it
About 1 in 3 children became completely free of seizures while taking the medication
Many children showed better alertness, communication, and less severe seizures
Children who continued taking the medication for an extended period had even fewer seizures; about 3 out of 4 seizures were prevented
Based on these encouraging results, the next phase of the EMBOLD study for children with SCN2A and SCN8A DEEs has begun
About the EMBOLD study
Purpose
To evaluate the safety and effectiveness of relutrigine (PRAX-562) in reducing seizures
Duration
Up to 26 weeks for part A, with an optional 48-week extension
At home - fully remote
Choose between fully remote, in-clinic, or combined participation
About relutrigine (PRAX-562)
Relutrigine is an investigational medicine designed to regulate sodium flow in brain cells more precisely, target overactive sodium channels causing seizures, and potentially offer better seizure control with fewer side effects. Relutrigine has been designed to maximize its effects against overactive sodium channels that are believed to cause seizure activity while minimizing the blocking of normal activity needed for healthy brain function.
SCN2A and SCN8A DEEs
DEE refers to a group of severe epilepsies that are characterized both by seizures, which are often drug resistant, as well as encephalopathy, which is a term used to describe significant developmental delay or even loss of developmental skills.
Why should my child participate?
The flexible study design allows study participation to be completed fully remote from your home, in the clinic at a study site, or a combination of both in-clinic and at-home visits- If your family chooses to attend in-clinic study visits, all expenses for travel, lodging, meals and any other costs associated with study participation will be paid for by the sponsor
- All participants will receive PRAX-562 during the study
- Option to participate in an open-label extension after the double-blind portion is complete
- In the open-label extension, all participants will have the opportunity to receive PRAX-562 for an additional 48 weeks after completion of part A. You can also continue to participate in a flexible manner
- PRAX-562 will be available after the open-label extension through an expanded access program for participants who showed clear benefits beyond previous therapeutic treatments
Your child may be able to participatea if they
- Are 2 through 18 years old
- Have received a diagnosis of
- SCN2A gene mutation with onset of seizures in the first 3 months of life; or,
- SCN8A gene mutation with seizures
- Have at least 8 motor seizures (seizures that involve movement) in the 4 weeks prior to screening
aAdditional criteria to be assessed at screening.
