RECRUITING

A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)

Conditions

SCN2A Encephalopathy SCN8A Encephalopathy Pediatric epilepsy NaV1.2 Voltage-Gated Sodium Channel SCN2A variant SCN8A variant Developmental and epileptic encephalopathy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is a Phase 2, double-blind, randomized clinical trial to explore the safety, tolerability, efficacy, and pharmacokinetics of PRAX-562 in pediatric participants who have seizures associated with early-onset SCN2A-DEE and SCN8A-DEE.

Official Title

A Phase 2,Double-Blind,Randomized Clinical Trial to Explore the Safety,Tolerability,Efficacy, and Pharmacokinetics of PRAX-562 in Pediatric Participants With Developmental and Epileptic Encephalopathies Followed by Open-Label Extension(OLE)

Quick Facts

Study Start:2023-08-02

Study Completion:2025-06-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05818553

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion Criteria
* Has a documented rare missense variant in SCN2A with onset of seizures occurring in the first three months of life or has a documented de novo (not observed in either parent) missense variant in SCN8A with onset of seizures occurring in the first six months of life. * Has a seizure frequency as follows: * At least 8 countable motor seizures in the 4 weeks immediately prior to Screening as reported by the parent/legal guardian or in the opinion of the investigator AND * At least 8 countable motor seizures during the 28 day Baseline Observation Period (during which seizure frequency is recorded in a daily seizure diary). * Additional inclusion criteria apply and will be assessed by the study team.	* Has any clinically significant or known pathogenic or likely pathogenic genetic variant other than in SCN2A and SCN8A or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder. * Has a documented, functionally characterized loss-of-function (LoF) missense variant or a presumed LoF variant (nonsense or frameshift variant) based on genetic testing and/or clinical evidence that prior exposure to a sodium channel blocker (SCB) medication worsened seizures. * Has 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening. * Additional exclusion criteria apply and will be assessed by the study team.

Inclusion Criteria

Exclusion Criteria

* Has a documented rare missense variant in SCN2A with onset of seizures occurring in the first three months of life or has a documented de novo (not observed in either parent) missense variant in SCN8A with onset of seizures occurring in the first six months of life.
* Has a seizure frequency as follows:
* At least 8 countable motor seizures in the 4 weeks immediately prior to Screening as reported by the parent/legal guardian or in the opinion of the investigator AND
* At least 8 countable motor seizures during the 28 day Baseline Observation Period (during which seizure frequency is recorded in a daily seizure diary).
* Additional inclusion criteria apply and will be assessed by the study team.

* Has any clinically significant or known pathogenic or likely pathogenic genetic variant other than in SCN2A and SCN8A or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder.
* Has a documented, functionally characterized loss-of-function (LoF) missense variant or a presumed LoF variant (nonsense or frameshift variant) based on genetic testing and/or clinical evidence that prior exposure to a sodium channel blocker (SCB) medication worsened seizures.
* Has 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening.
* Additional exclusion criteria apply and will be assessed by the study team.

Contacts and Locations

Study Contact

Head of Pharmacovigilance

CONTACT

617-300-8460

clinicaltrials@praxismedicines.com

Principal Investigator

Medical Director

STUDY_DIRECTOR

Praxis Precision Medicines

Study Locations (Sites)

Praxis Research Site

Atlanta, Georgia, 30329

United States

Praxis Research Site

Chicago, Illinois, 60611

United States

Praxis Research Site

Boston, Massachusetts, 02115

United States

Praxis Research Site

Tacoma, Washington, 98405

United States

Collaborators and Investigators

Sponsor: Praxis Precision Medicines

Medical Director, STUDY_DIRECTOR, Praxis Precision Medicines

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-08-02

Study Completion Date2025-06-30

Study Record Updates

Study Start Date2023-08-02

Study Completion Date2025-06-30

Terms related to this study

Keywords Provided by Researchers

Pediatric epilepsy
NaV1.2 Voltage-Gated Sodium Channel
SCN2A variant
SCN8A variant
Developmental and epileptic encephalopathy

Additional Relevant MeSH Terms

SCN2A Encephalopathy
SCN8A Encephalopathy