About the EMBOLD Study

The PRAX-562-221 EMBOLD Study is advancing a clinical stage, novel treatment built for children with early-onset SCN2A and SCN8A Developmental and Epileptic Encephalopathies (DEE).

PRAX-562 is a novel, clinical-stage medication built for children with SCN2A-DEE and SCN8A-DEE, with the potential to be a best-in-class sodium channel blocker. Many current treatments target sodium channels, but do not target persistent sodium current potently and selectively. PRAX-562 is designed to precisely target persistent sodium current, which is believed to be a critical driver of pathological hyperexcitability and seizures in children with SCN2A-DEE and SCN8A-DEE. PRAX-562 preclinical data has shown an unprecedented therapeutic window that may offer a more effective and tolerable option for children with DEEs, with potential for more effective seizure control and reduced side effects.

The EMBOLD Study is a randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate the safety, tolerability, efficacy (motor seizure frequency) and pharmacokinetics (PK) of PRAX-562 in children aged 2 to 18 years with DEEs, followed by an open-label extension.

Why should my child participate?

*         PRAX-562 was specifically designed for children with rare epilepsies

*         PRAX-562 is a once daily investigational drug administered as a liquid suspension, orally or through a gastrostomy tube (G-tube). This reduces medication administration burden in some participants.

*         Pre-clinical studies indicate that PRAX-562 has a long half-life. This means that medication needs to be given just once a day and the levels may stay more consistent over a longer period.

*         Animal studies showed PRAX-562 significantly reduced seizures, up to complete inhibition. 

*         Studies conducted in healthy human volunteers showed PRAX-562 was generally well-tolerated.

It is important to note that PRAX-562 is an investigational drug, which means that it has not been approved by any regulatory or health authorities, including the U.S. Food and Drug Administration (FDA). Participation in clinical research studies is completely voluntary and you may end participation for your child at any time.

 

About Participating in the EMBOLD Study

*         All participants will receive PRAX-562 at some timepoint during the study

*         The study will last approximately 26 weeks with about 11 study visits at the study clinic, your home, or over the phone

*        There will be an optional open-label extension period during which all participants will receive PRAX-562 for an additional 48 weeks

*         Travel may be required for some participants, all expenses related to study participation will be covered by the sponsor.

 

 

Your child may be able to take part if they:

 

*Are 2 through 18 years old

 

*Have either:

* A documented SCN2A mutation diagnosis with onset of seizures in the first three months of life

OR

* A documented SCN8A mutation diagnosis with onset of seizures in the first six months of life

 

*Have at least 8 motor seizures in the four weeks prior to screening


Note: motor seizures are defined as tonic, tonic-clonic, atonic/drop attacks, focal with secondary generalization or focal with motor symptoms.

 

Schedule a call with a Nurse Patient Navigator

 

If you think your child may qualify for the EMBOLD study and would like to speak to a Nurse Patient Navigator to learn more, please schedule a pre-screening appointment by using the calendar function below.

 

If you don't think your child qualifies for the EMBOLD study, you may still schedule a call with a Nurse Patient Navigator to discuss your child's specific situation.

 

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