Recruiting
Phase 3

Elsunersen

Sponsor:

Praxis Precision Medicines

Code:

NCT07019922

Conditions

Epileptic Encephalopathy

SCN2A Encephalopathy

Eligibility Criteria

Sex: All

Age: 0 - 18

Healthy Volunteers: Not accepted

Interventions

1mg elsunersen

0.5mg elsunersen

Study Details

Brief summary:

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

Conditions

Epileptic Encephalopathy

SCN2A Encephalopathy

Study ID

NCT07019922

Start date

Aug 13, 2025

Status verified date

Jul, 2026

Completion date

Dec, 2028

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 18

Healthy Volunteers: Not accepted

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

Enrollment

40 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1 Arm 1: Open-Label Treatment Period

Open-label elsunersen

experimental: Cohort 2: Open-Label Treatment Period

Open-label elsunersen

experimental: Cohort 3: Open-Label Treatment Period

Open-label elsunersen

Interventions

1mg elsunersen

24 weeks every 4 weeks intrathecally

0.5mg elsunersen

24 weeks every 4 weeks intrathecally

Primary outcome measure

  • To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE [ Time Frame: 24 weeks ]

Central Contacts and Locations

Central contacts

Head of Pharmacovigilance

617-300-8460clinicaltrials@praxismedicines.com

Locations

Praxis Research Site

Recruiting

San Diego, California, United States, 92123

Praxis Research Site

Recruiting

Aurora, Colorado, United States, 80045

Praxis Research Site

Recruiting

Chicago, Illinois, United States, 60612

Praxis Research Site

Recruiting

Cincinnati, Ohio, United States, 45229

Praxis Research Site

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Praxis Research Site

Recruiting

Memphis, Tennessee, United States, 38105

More Information

Sponsor

Praxis Precision Medicines

Last update posted

Aug 3, 2026

Last verified

Jul, 2026

Keywords

  • epilepsis
  • early onset scn2a
  • epileptic encephalopathy
  • scn2a
  • gain of function
  • seizure

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Praxis Precision Medicines on 2026-08-03.