NCT07019922

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

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at below P25 for phase_3

Timeline
28mo left

Started Aug 2025

Typical duration for phase_3

Geographic Reach
4 countries

10 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress29%
Aug 2025Dec 2028

First Submitted

Initial submission to the registry

June 5, 2025

Completed
8 days until next milestone

First Posted

Study publicly available on registry

June 13, 2025

Completed
2 months until next milestone

Study Start

First participant enrolled

August 13, 2025

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2026

Completed
2.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2028

Expected
Last Updated

May 27, 2026

Status Verified

May 1, 2026

Enrollment Period

10 months

First QC Date

June 5, 2025

Last Update Submit

May 22, 2026

Conditions

Epileptic EncephalopathySCN2A Encephalopathy

Keywords

epilepsisearly onset scn2aepileptic encephalopathyscn2again of functionseizure

Outcome Measures

Primary Outcomes (1)

  • 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

    24 weeks

Secondary Outcomes (8)

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

    24 weeks

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

    24 weeks

  • CGI-S change from baseline

    24 weeks

  • CGI subdomain scores at each postdose time point

    24 weeks

  • CgGI-S from baseline

    24 weeks

  • +3 more secondary outcomes

Study Arms (3)

Cohort 1 Arm 1: Open-Label Treatment Period

EXPERIMENTAL

Open-label elsunersen

Drug: 1mg elsunersen

Cohort 2: Open-Label Treatment Period

EXPERIMENTAL

Open-label elsunersen

Drug: 1mg elsunersen

Cohort 3: Open-Label Treatment Period

EXPERIMENTAL

Open-label elsunersen

Drug: 0.5mg elsunersen

Interventions

1mg elsunersenDRUG

24 weeks every 4 weeks intrathecally

Cohort 1 Arm 1: Open-Label Treatment PeriodCohort 2: Open-Label Treatment Period
0.5mg elsunersenDRUG

24 weeks every 4 weeks intrathecally

Cohort 3: Open-Label Treatment Period

Eligibility Criteria

Age1 Day - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Praxis Research Site

San Diego, California, 92123, United States

RECRUITING

Praxis Research Site

Aurora, Colorado, 80045, United States

RECRUITING

Praxis Research Site

Chicago, Illinois, 60612, United States

RECRUITING

Praxis Research Site

Cincinnati, Ohio, 45229, United States

RECRUITING

Praxis Research Site

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Praxis Research Site

Memphis, Tennessee, 38105, United States

RECRUITING

Praxis Research Site

Porto Alegre, 90035, Brazil

WITHDRAWN

Praxis Research Site

São Paulo, 05403, Brazil

WITHDRAWN

Praxis Research Site

Bonn, Bonn, 53127, Germany

RECRUITING

Praxis Research Site

Rome, 00165, Italy

RECRUITING

MeSH Terms

Conditions

EpilepsySeizures

Condition Hierarchy (Ancestors)

Brain DiseasesCentral Nervous System DiseasesNervous System DiseasesNeurologic ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Study Officials

  • Medical Director

    Praxis Precision Medicines

    STUDY DIRECTOR

Central Study Contacts

Head of Pharmacovigilance

CONTACT

617-300-8460clinicaltrials@praxismedicines.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: Specific milestones have to be met in Cohort 1 for enrollment to open in Cohort 2 and the same will apply to open Cohort 3.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 5, 2025

First Posted

June 13, 2025

Study Start

August 13, 2025

Primary Completion

June 1, 2026

Study Completion (Estimated)

December 1, 2028

Last Updated

May 27, 2026

Record last verified: 2026-05

Locations

US(6)BR(2)DE(1)IT(1)