The EMBOLD Study
for children with early-onset SCN2A and SCN8A Developmental and Epileptic Encephalopathies (DEE)
About the EMBOLD Study
The EMBOLD study is now offering a fully in-home participation option in which all study visits can be done from your home. We know traveling can be difficult for this patient population, so we are bringing the trial to you.
The main purpose of this study is to learn about the effects of the study drug, PRAX-562, by assessing the safety and impact on numbers of seizures.
Part A of the study will last up to 26 weeks and consist of 11 study visits. Your family will have the option to complete these study visits completely remote from your home, in clinic at a study site or a combination of both in-clinic and at-home visits. If PRAX-562 shows benefit in Part A, all participants will have the option to enroll into an open-label extension for up to an additional 48 weeks. In the open-label extension, all participants will continue to receive PRAX-562 and you can continue to participate in a flexible manner, in clinic and/or at home. PRAX-562 will be available after the open-label extension through an expanded access program for participants who showed clear benefit beyond previous therapeutic treatments.
What is PRAX-562 and how is it potentially different from currently available therapies?
PRAX-562 is an investigational medicine that has been designed to more precisely regulate the flow of sodium in and out the cells in the brain. Currently available sodium channel blockers work to block the flow of sodium across all sodium channel types. PRAX-562 has been designed to maximize its effects against overactive sodium channels that are believed to cause seizure activity while minimizing the blocking of the normal activity needed for healthy brain functioning. We believe this precision approach may offer greater seizure control with reduced side effects.
Why should my child participate?
Flexible study design allows study participation to be completed fully remote from your home, in 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 benefit beyond previous therapeutic treatments.
Your child may be able to participate if they:
Are 2 through 18 years old
Have received a diagnosis of:
SCN2A gene mutation with onset of seizures in the first three months of life; or,
SCN8A gene mutation with seizures
Have at least eight motor seizures (seizures that involve movement) in the four weeks prior to screening
Additional criteria to be assessed at screening
How does my child participate?
Connect with a Nurse Patient Navigator by following the instructions below. The Nurse Navigator will confirm your child’s eligibility and connect you with an EMBOLD study site.
1. Click the button below to fill out the form with your contact information
2. Select a day and time to speak with the EMBOLD Nurse Navigator.
3. Once complete, click ‘Submit Request’
By clicking the above button and filling out the form, you consent to providing this information to Praxis Precision Medicines, Inc. (Praxis) and its employees, affiliates, and service providers. You understand that your information will be used to enable Praxis to engage in prescreening for the research study. You permit Praxis to share your information with third parties, including the Sites, Clinical Research Organizations, the Eligibility Review Board, and regulatory or public health agencies, in order to evaluate your eligibility for the research study.
Additionally, with this consent, Praxis will retain such information to provide you with updates, newsletters and communication related to Praxis’ ongoing studies and business updates.