ACTIVE_NOT_RECRUITING

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

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Conditions

SCN2A EncephalopathySCN8A EncephalopathyPediatric epilepsyNaV1.2 Voltage-Gated Sodium ChannelSCN2A variantSCN8A variantDevelopmental 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

A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (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:2027-03-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_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:1 Year to 18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Has a documented variant in SCN2A with onset of seizures occurring in the first 3 months of life or has a diagnosis of SCN8A-DEE supported by both clinical and genetic findings.
  2. * Has a seizure frequency as follows:
  3. * 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 as documented in medical notes.
  1. * 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.
  2. * 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.
  3. * Has 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening.
  4. * Additional exclusion criteria apply and will be assessed by the study team.

Contacts and Locations

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
Minneapolis, Minnesota, 55113
United States
Praxis Research Site
Hackensack, New Jersey, 07601
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 Date2027-03-01

Study Record Updates

Study Start Date2023-08-02
Study Completion Date2027-03-01

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