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

ConditionsEpileptic EncephalopathySCN2A Encephalopathy
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age1-18
SponsorPraxis Precision Medicines

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • 1mg elsunersen

    Drug

    24 weeks every 4 weeks intrathecally

  • 0.5mg elsunersen

    Drug

    24 weeks every 4 weeks intrathecally

Treatment groups

40 Participants
are divided into 3 treatment groups
Group A: Cohort 1 Arm 1: Open-Label Treatment PeriodExperimental treatment 1 intervention
Group B: Cohort 2: Open-Label Treatment PeriodExperimental treatment 1 intervention
Group C: Cohort 3: Open-Label Treatment PeriodExperimental treatment 1 intervention

Trial outcomes

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

Time frame
24 weeks

Secondary outcomes

1

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

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

Time frame
24 weeks
2

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

Change in motor seizure-free days from baseline

Time frame
24 weeks
3

CGI-S change from baseline

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).

Time frame
24 weeks
4

CGI subdomain scores at each postdose time point

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

Time frame
24 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Praxis Precision Medicines

Lead sponsor

Source ClinicalTrials.gov
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