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

Purpose

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 Encephalopathy
SCN2A Encephalopathy

Eligibility

Eligible Ages: Between 1 Day and 18 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

Inclusion 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.

Exclusion Criteria

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.

Study Design

Phase: Phase 3
Study Type: Interventional
Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Specific milestones have to be met in Cohort 1 for enrollment to open in Cohort 2 and the same will apply to open Cohort 3.
Primary Purpose: Treatment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Cohort 1 is the only masked cohort (40 participants). Cohort 2 and Cohort 3 are both open-label and will have 5 participants each.

Arm Groups

Arm	Description	Assigned Intervention
Experimental Cohort 1 Arm 1: Double-Blind Treatment Period	Double-blind treatment period elsunersen	Drug: 1mg elsunersen 24 weeks every 4 weeks intrathecally
Sham Comparator Cohort 1 Arm 2: Double-Blind Treatment Period	Double-blind sham-procedure	Procedure: sham procedure 24 weeks of sham-procedure every 4 weeks
Experimental Cohort 2: Open-Label Treatment Period	Open-label elsunersen	Drug: 1mg elsunersen 24 weeks every 4 weeks intrathecally
Experimental Cohort 3: Open-Label Treatment Period	Open-label elsunersen	Drug: 0.5mg elsunersen 24 weeks every 4 weeks intrathecally

Recruiting Locations

Praxis Research Site
San Diego, California 92123

Praxis Research Site
Chicago, Illinois 60612

More Details

NCT ID: NCT07019922
Status: Recruiting
Sponsor: Praxis Precision Medicines

Study Contact

Head of Pharmacovigilance
617-300-8460
clinicaltrials@praxismedicines.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.