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

Part of paid clinical trials in San Diego, California.

Sponsor
Praxis Precision Medicines
Study ID
NCT07019922
Phase
PHASE3
Status
Recruiting
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Conditions

  • Epileptic Encephalopathy
  • SCN2A Encephalopathy

Eligibility Criteria

Sex
ALL
Age
1 Day - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • 1mg elsunersen — DRUG
    24 weeks every 4 weeks intrathecally
  • 0.5mg elsunersen — DRUG
    24 weeks every 4 weeks intrathecally

Study Details

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

Key Dates

Start date
Aug 13, 2025
Status verified
May 2026
Primary completion
Jun 30, 2026
Completion
Dec 31, 2028

Study Design

Enrollment
40 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

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

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

Locations (6)

FacilityCityStateZIPSite coordinators
Praxis Research SiteSan DiegoCalifornia92123-
Praxis Research SiteAuroraColorado80045-
Praxis Research SiteChicagoIllinois60612-
Praxis Research SiteCincinnatiOhio45229-
Praxis Research SitePhiladelphiaPennsylvania19104-
Praxis Research SiteMemphisTennessee38105-

Find similar trials in San Diego, CA

By research site
Praxis Research Site· San Diego, CAPraxis Research Site· Aurora, COPraxis Research Site· Chicago, ILPraxis Research Site· Cincinnati, OHPraxis Research Site· Philadelphia, PAPraxis Research Site· Memphis, TN

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