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

Phase 3RecruitingNCT07019922

Praxis Precision Medicines50 enrolled

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Epileptic Encephalopathy SCN2A Encephalopathy

Interventions

1mg elsunersenDRUG

24 weeks every 4 weeks intrathecally

sham procedurePROCEDURE

24 weeks of sham-procedure every 4 weeks

0.5mg elsunersenDRUG

24 weeks every 4 weeks intrathecally

Eligibility

Sex: ALLMin age: 1 DayMax age: 18 Years

Medical Language ↔ Plain English

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.

Locations (2)

Praxis Research Site

San Diego, California, United States

RECRUITING

Praxis Research Site

Chicago, Illinois, United States

RECRUITING

Outcomes

Primary Outcomes

To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE

Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks

Time frame: 24 weeks

Secondary Outcomes

To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE

Responder rate - defined as a ≥50% reduction in monthly seizure frequency from baseline compared to treatment after 24 weeks

Time frame: 24 weeks

To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE

Change in motor seizure-free days from baseline

Time frame: 24 weeks

CGI-S change from baseline

CGI-S assesses the clinician's impression of the participant's current illness state. The clinician should use his/her total clinical experience with this patient population and rate the current severity on a 7-point scale from 1 (normal, not at all ill) to 7 (among the most extremely ill patients).

Time frame: 24 weeks

CGI subdomain scores at each postdose time point

Clinical Global Impression-Improvement (CGI-I) subdomains scores at each postdose time point

Time frame: 24 weeks

CgGI-S from baseline

Caregiver Global Impression-Severity (CgGI-S) at baseline compared to treatment after 24 weeks

Time frame: 24 weeks

CgGI-I subdomain scores at each postdose time point

Caregiver Global Impression-Improvement (CgGI I) subdomains scores at each postdose time point

Central Contacts

Head of Pharmacovigilance

CONTACT

617-300-8460 clinicaltrials@praxismedicines.com

Data from ClinicalTrials.gov

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