Status:

RECRUITING

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

Lead Sponsor:

Praxis Precision Medicines

Conditions:

Epileptic Encephalopathy

SCN2A Encephalopathy

Eligibility:

All Genders

1-18 years

Phase:

PHASE3

Brief Summary

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 a...

Eligibility Criteria

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.

Key Trial Info

Start Date :

August 13 2025

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 1 2028

Estimated Enrollment :

50 Patients enrolled

Trial Details

Trial ID

NCT07019922

Start Date

August 13 2025

End Date

December 1 2028

Last Update

October 2 2025

Active Locations (2)

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Page 1 of 1 (2 locations)

1

Praxis Research Site

San Diego, California, United States, 92123

2

Praxis Research Site

Chicago, Illinois, United States, 60612

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