RECRUITING

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

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

Conditions

Epileptic EncephalopathySCN2A Encephalopathy
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Related Conditions:

Epileptic EncephalopathySCN2A Encephalopathy

Study Overview

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Study Details

Study overview

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

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

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

Condition
Epileptic Encephalopathy
Intervention / Treatment

-

Contacts and Locations

Chicago

Praxis Research Site, Chicago, Illinois, United States, 60612

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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Has a documented Gain of Function SCN2A variant confirmed through genetic testing.
  • * Has onset of seizures prior to 3 months of age.
  • * Seizure frequency of 4 or more countable motor seizures per 28-day during the Baseline Observation Period.
  • * 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.
  • * Has bone, spine (eg, kyphosis, scoliosis), bleeding, or other disorder.
  • * 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.
  • * Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial.

Ages Eligible for Study

1 Day to 18 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Praxis Precision Medicines,

Medical Director, STUDY_DIRECTOR, Praxis Precision Medicines

Study Record Dates

2028-12