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

Praxis Precision Medicines

Status and phase: Enrolling; Phase 3

Conditions: Epileptic Encephalopathy; SCN2A Encephalopathy

Treatments: Drug: 1mg elsunersen; Drug: 0.5mg elsunersen; Procedure: sham procedure

Study type: Interventional

Funder types: Industry

Identifiers

NCT07019922

PRAX-222-311

Details and patient eligibility

About

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

Enrollment: 50 estimated patients

Sex: All

Ages: 1 day to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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50 participants in 4 patient groups

Cohort 1 Arm 1: Double-Blind Treatment Period

Experimental group

Description:

Double-blind treatment period elsunersen

Treatment:

Drug: 1mg elsunersen

Cohort 1 Arm 2: Double-Blind Treatment Period

Sham Comparator group

Description:

Double-blind sham-procedure

Treatment:

Procedure: sham procedure

Cohort 2: Open-Label Treatment Period

Experimental group

Description:

Open-label elsunersen

Treatment:

Drug: 1mg elsunersen

Cohort 3: Open-Label Treatment Period

Experimental group

Description:

Open-label elsunersen

Treatment:

Drug: 0.5mg elsunersen