RECRUITING

A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety

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Conditions

Epileptic EncephalopathySCN2A Encephalopathyepilepsisearly onset scn2ascn2again of functionseizure

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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 Randomized, Multi-Center, Double-Blind, Sham-Procedure-Controlled Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy

Official Title

A Randomized, Multi-Center, Double-Blind, Sham-Procedure-Controlled Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy

Quick Facts

Study Start:2025-08-13
Study Completion:2028-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT07019922

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 Day to 18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Has a documented Gain of Function SCN2A variant confirmed through genetic testing.
  2. * Has onset of seizures prior to 3 months of age.
  3. * Seizure frequency of 4 or more countable motor seizures per 28-day during the Baseline Observation Period.
  1. * Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder.
  2. * Has bone, spine (eg, kyphosis, scoliosis), bleeding, or other disorder.
  3. * Has received any experimental or investigational drug, device, or other therapy within 30 days or 5 half-lives (whichever is longer) prior to Screening, including any prior use of gene therapy.
  4. * Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial.

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
Chicago, Illinois, 60612
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 Date2025-08-13
Study Completion Date2028-12

Study Record Updates

Study Start Date2025-08-13
Study Completion Date2028-12

Terms related to this study

Keywords Provided by Researchers

  • epilepsis
  • early onset scn2a
  • epileptic encephalopathy
  • scn2a
  • gain of function
  • seizure

Additional Relevant MeSH Terms

  • Epileptic Encephalopathy
  • SCN2A Encephalopathy