NCT07019922招募中3 期
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
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 11
- 主要终点
- To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Cohort 1 is the only masked cohort (40 participants). Cohort 2 and Cohort 3 are both open-label and will have 5 participants each.
入排标准
- 年龄范围
- 1 Day 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Cohort 1 Arm 1: Open-Label Treatment Period
Experimental
Open-label elsunersen
干预措施: 1mg elsunersen (Drug)
Cohort 3: Open-Label Treatment Period
Experimental
Open-label elsunersen
干预措施: 0.5mg elsunersen (Drug)
Cohort 2: Open-Label Treatment Period
Experimental
Open-label elsunersen
干预措施: 1mg elsunersen (Drug)
结局指标
主要结局
To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE
时间窗: 24 weeks
Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks
次要结局
- To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE(24 weeks)
- Sleep assessment scores from baseline(24 weeks)
- CgGI-I subdomain scores at each postdose time point(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)
- To evaluate the safety and tolerability of elsunersen in participants with early-onset SCN2A DEE(24 weeks)
研究者
研究点 (11)
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