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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-08-13

Study Completion Date

2028-12-31

Brief Summary

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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

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Detailed Description

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Conditions

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Epileptic Encephalopathy SCN2A Encephalopathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

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 Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Cohort 1 is the only masked cohort (40 participants). Cohort 2 and Cohort 3 are both open-label and will have 5 participants each.

Study Groups

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Cohort 1 Arm 1: Double-Blind Treatment Period

Double-blind treatment period elsunersen

Group Type EXPERIMENTAL

1mg elsunersen

Intervention Type DRUG

24 weeks every 4 weeks intrathecally

Cohort 1 Arm 2: Double-Blind Treatment Period

Double-blind sham-procedure

Group Type SHAM_COMPARATOR

sham procedure

Intervention Type PROCEDURE

24 weeks of sham-procedure every 4 weeks

Cohort 2: Open-Label Treatment Period

Open-label elsunersen

Group Type EXPERIMENTAL

1mg elsunersen

Intervention Type DRUG

24 weeks every 4 weeks intrathecally

Cohort 3: Open-Label Treatment Period

Open-label elsunersen

Group Type EXPERIMENTAL

0.5mg elsunersen

Intervention Type DRUG

24 weeks every 4 weeks intrathecally

Interventions

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1mg elsunersen

24 weeks every 4 weeks intrathecally

Intervention Type DRUG

sham procedure

24 weeks of sham-procedure every 4 weeks

Intervention Type PROCEDURE

0.5mg elsunersen

24 weeks every 4 weeks intrathecally

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

1 Day

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No