FAQs

The current DEE studies being conducted by Praxis Precision Medicines are EMBOLD (relutrigine), EMBRAVE 3 (elsunersen), and EMERALD (relutrigine).

After you complete the prescreening questions, our system will generate the study or studies your child is prequalified for. You will then be asked to schedule a call to speak with a nurse navigator who will ask further questions to determine if your child is a candidate to be referred to one of our study sites.

A motor seizure occurs when you notice any sudden, uncontrolled movements like jerking or stiffening of the body or any drooping of the body.

A double-blind study means neither you nor the doctors know if your child is receiving the investigational medication or a placebo. This helps keep the results fair and unbiased.

A placebo is something that looks like real medication but doesn’t have any active ingredients in it. It will not harm your child. It’s used in research to help understand how well an investigational medicine or treatment works.

Open label means only the investigational medicine is being given; there is no placebo or control. All 3 of our DEE studies include an option to participate in an open-label extension following completion of the initial controlled study.

Your child will still see their current neurologist and will be taking the investigational medicine alongside their current epilepsy treatment, if eligible. This study team will not make any changes regarding your child’s current epilepsy care.

Following completion of the open-label extension, the investigational medicine will be made available through an expanded access program for participants who showed clear benefits of this treatment based on local country requirements.

Yes; however, they must have stable settings or parameters 1 month prior to screening.

The call will cover an overview of the study, as well as questions about your child’s medical history and other important details to determine if your child is prequalified to participate. The call should take between 30 and 60 minutes, if translation is required.

Yes! Travel, lodging, and other related costs will be covered by Praxis Precision Medicines. Families will not have to pay any study-related travel expenses.

Yes. We will support each family however we can, on a case-by-case basis.

Yes! We provide assistance and coordination with relocation to any of our study sites.

Participation in research studies is entirely voluntary and you may withdraw your child’s participation at any time. If you decide to withdraw your child early, you will be asked to notify the staff before doing so and have one final safety visit.

Your child’s safety is our top priority. In all 3 studies, there are several steps taken to closely monitor your child’s health throughout. Regular lab tests are done to check how your child’s body is responding to the investigational medication and watch for any side effects. During the observational period, specific tests will be done to help track your child’s baseline health before starting treatment.

The investigational medicine route is intrathecal, which means it is given through a small injection into the fluid around the spine. It is similar to how an epidural or spinal tap is done. This will be administered at the study site by trained professionals every 4 weeks.

Elsunersen is an antisense oligonucleotide (ASO). An ASO is a type of medicine from small pieces of genetic material (similar to DNA) that can help fix how certain genes work. It works by attaching to specific messages (mRNA) in the body that tell cells how to make proteins. If a gene is causing a problem, an ASO can block or change that message, helping to reduce or stop the problem.

Elsunersen has been previously administered to pediatric patients, showing consistent and significant seizure reduction in every patient treated, among other developmental benefits.

We are conducting this study on participants aged 0 to 18 years; as long as they are above 37 weeks gestational age, they may be eligible. The first part of the study will enroll patients aged 2 to 18 years old, followed later with potential to enroll patients aged 0 to 2 years old.

The first part of the EMBRAVE 3 study will be approximately 24 weeks with an option to continue into a 24-week open-label extension period.

If your child is experiencing benefit, they will have the opportunity to remain on study drug through an expanded access program as allowed.

As this study requires intrathecal administration, your child will need to have an in-person appointment once a month for the duration of the study; however, any appointments in between can be completed remotely from your home.

Please have a copy of your child’s genetic report confirming SCN2A genetic mutation along with the DNA change and protein change.

The EMERALD study will last up to 24 weeks with an option to continue into a 32-week open-label extension period.

If your child is experiencing benefit, they will have the opportunity to remain on study drug through an expanded access program as allowed.

Relutrigine is an investigational medicine and can be administered orally by mouth or through a G/J-tube. Administration is once daily.

If you are located in the United States or consent to being relocated to the United States, your child may participate in the study remotely, in person, or through a combination of both, whichever your family prefers.

Yes, there is a placebo arm in this study. Your child will have a 50% (1 in 2) chance of receiving the investigational medicine or placebo. This is used to determine the efficacy of the investigational medicine by our research team; however, all participants in the study will receive the active investigational medicine at some point during the study.

Relutrigine has been previously administered to pediatric patients, showing consistent and significant seizure reduction in every patient treated, amongst other developmental benefits.