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NCT Number: NCT07019922

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

A Multi-Center, Single-Arm 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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Key information

Conditions

Epileptic Encephalopathy Brain Diseases Central Nervous System Diseases Epilepsy Nervous System Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms SCN2A Encephalopathy Seizures Signs and Symptoms

Age range

1 day–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Praxis Research Site, Porto Alegre, Brazil

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

1mg elsunersen

Drug

24 weeks every 4 weeks intrathecally

0.5mg elsunersen

Drug

24 weeks every 4 weeks intrathecally

Primary outcomes

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

    Time frame: 24 weeks

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

Secondary outcomes

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

    Time frame: 24 weeks

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

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

    Time frame: 24 weeks

    Change in motor seizure-free days from baseline

  3. CGI-S change from baseline

    Time frame: 24 weeks

    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).

  4. CGI subdomain scores at each postdose time point

    Time frame: 24 weeks

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

  5. CgGI-S from baseline

    Time frame: 24 weeks

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

  6. CgGI-I subdomain scores at each postdose time point

    Time frame: 24 weeks

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

  7. Sleep assessment scores from baseline

    Time frame: 24 weeks

    Sleep assessment scores at baseline compared to each postdose time point

  8. To evaluate the safety and tolerability of elsunersen in participants with early-onset SCN2A DEE

    Time frame: 24 weeks

    Incidence and severity of treatment-emergent adverse events (TEAEs)

Study contacts

Contact information is provided by the study sponsor or research team.

Head of Pharmacovigilance

CONTACT

[email protected]

617-300-8460

Sponsors and collaborators

Lead sponsor

Praxis Precision Medicines

Industry

Registry information

Official study title

A Multi-Center, Single-Arm Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy

Important dates

Study start
2025
Primary completion
2026
Study completion
2028
First posted
Jun 13, 2025
Registry last updated
May 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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