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for children with early-onset SCN2A and SCN8A Developmental and Epileptic Encephalopathies (DEE)

The EMBOLD Study

Topline Data from the first part of Embold Study in
SCN2A and SCN8A Developmental and Epileptic Encephalopathies, resulted in:

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  • Placebo-adjusted monthly motor seizure reduction of 46% during double-blind period

  • Over 30% of patients achieved seizure freedom status while on relutrigine

  • Meaningful gains observed in alertness, communication and seizure severity

  • 75% reduction in median seizure rate seen for patients in the long-term extension

  • Registrational phase of the EMBOLD study for SCN2A and SCN8A initiated

Relutrigine EMBOLD Readout_Final.jpg
Relutrigine EMBOLD Readout_Final_v2.jpg
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

Connect with a Nurse Patient Navigator to learn more about the next study. The Nurse Navigator will confirm your child’s eligibility and connect you with an EMBOLD study site.

 


 

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.

 

Praxis will retain your personal data in accordance with its regulatory record keeping requirements or for as long as we have a legitimate legal or business need to do so. You understand that you can withdraw your consent at any time by contacting Privacy@praxismedicines.com. For more on our privacy practices and your privacy rights, including the right to withdraw your consent, please refer to our Privacy Policy at https://praxismedicines.com/privacy-policy/.

 

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