PRAX-222-111 EMBRAVE Study Pre-screen

The EMBRAVE study will evaluate PRAX-222, the first potential disease-modifying treatment for early-onset SCN2A Developmental and Epileptic Encephalopathy (DEE)

About PRAX-222

PRAX-222 is an investigational antisense oligonucleotide (ASO) with the potential to be the first disease-modifying treatment for early-onset SCN2A Developmental and Epileptic Encephalopathy (DEE).

An ASO is a short, synthetic segment of genetic sequence designed to correct the malfunction in a specific gene. In animal studies, PRAX-222 was shown to significantly reduce seizures, normalize developmental curves and improve survival. By targeting the underlying genetic cause of disease, PRAX-222 has demonstrated potential to go beyond seizures to treating other symptoms of the disease.

About the EMBRAVE Study (Part 1)

Part 1 of the EMBRAVE study is designed to determine the safety and tolerability of PRAX-222 in the treatment of early-onset SCN2A DEE. In Part 1 of the study, all participants will receive PRAX-222 for up to 13 weeks.

It is important to note that PRAX-222 is an investigational drug, which means that it has not been approved for the treatment of SCN2A DEE 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.

Children who meet the below Key Inclusion Criteria may be eligible to participate:

* Resides in the United States of America

* Documented SCN2A variant through genetic testing

* Had seizure onset within the first 3 months of life.

* Currently taking 2 or less sodium channel blocking anti-epileptic drugs.

** Note: For the purposes of this clinical trial, sodium channel blocking ASMs will include carbamazepine, eslicarbazepine, lacosamide, lamotrigine, oxcarbazepine, phenytoin and rufinamide.

* Male or female between the ages of 2 to 18 years of age

* Has at least 8 motor seizures in the 4 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.

Speak to a nurse about the EMBRAVE Study
To schedule call with a Praxis Nurse Patient Navigator:

Please review and complete the consent checkbox below.
Once you check the box a calendar will open, and you can select a day and time to speak with the EMBRAVE nurse navigator.

By checking this box, 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.

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