Recruiting NCT07019922
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: 1mg elsunersen, 0.5mg elsunersen.
- Who it may be relevant to
- Registry conditions: Epileptic Encephalopathy, SCN2A Encephalopathy. Basic parameters: 1 Day — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Brazil, Germany, Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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
Interventions
- Drug 1mg elsunersen
24 weeks every 4 weeks intrathecally - Drug 0.5mg elsunersen
24 weeks every 4 weeks intrathecally
Primary outcome measures
- To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE [Time frame: 24 weeks]
Secondary outcome measures (8)
- To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE [Time frame: 24 weeks]
- To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE [Time frame: 24 weeks]
- CGI-S change from baseline [Time frame: 24 weeks]
- CGI subdomain scores at each postdose time point [Time frame: 24 weeks]
- CgGI-S from baseline [Time frame: 24 weeks]
- CgGI-I subdomain scores at each postdose time point [Time frame: 24 weeks]
- Sleep assessment scores from baseline [Time frame: 24 weeks]
- To evaluate the safety and tolerability of elsunersen in participants with early-onset SCN2A DEE [Time frame: 24 weeks]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 6 centers
- Praxis Research Site — San Diego
- Praxis Research Site — Aurora
- Praxis Research Site — Chicago
- Praxis Research Site — Cincinnati
- Praxis Research Site — Philadelphia
- Praxis Research Site — Memphis
Brazil · 2 centers
- Praxis Research Site — Porto Alegre
- Praxis Research Site — São Paulo
Germany · 1 center
- Praxis Research Site — Bonn
Italy · 1 center
- Praxis Research Site — Rome
Identifiers
NCT: NCT07019922 · PRAX-222-311