Multi-site, Prospective, Open-label, Long-term, Flexible Dose, Interventional Study to Evaluate the Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 9
- 主要终点
- Number of Participants With Adverse Events as a Measure of Safety and Tolerability
研究概览
简要总结
To investigate the long-term safety and tolerability of clobazam when administered for 1 year as adjunctive therapy in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient has a diagnosis of Dravet Syndrome supported by:
- •onset of seizures in the first year of life
- •history of fever-induced prolonged seizures as determined by the Investigator
- •these may include prolonged (approximately 15 minutes or longer) hemi-clonic seizures
- •multiple seizure types which may include:
- •generalised tonic-clonic (required for inclusion)
- •clonic (required for inclusion)
- •myoclonic jerks/seizures
- •history of normal development prior to seizure onset followed by development delay or regression after seizure onset
- •abnormal EEG consistent with Dravet Syndrome
- •The patient is currently receiving a stable dose of clobazam of at least 0.5 mg/kg/day (maximum 20 mg/day) for at least 3 months
- •Other protocol-defined inclusion and exclusion criteria may apply.
排除标准
- 未提供
研究组 & 干预措施
Clobazam
A maximum of 2.0 mg/kg/day (maximum 80 mg/day) twice daily (BID); clobazam oral suspension (2.5 mg/mL) or clobazam scored tablets (10 mg), orally
干预措施: Clobazam (Drug)
结局指标
主要结局
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
时间窗: Up to Day 390
Change in Behavioural, Neurocognitive Measures Using Vineland Adaptive Behaviour Scale (VABS)
时间窗: Baseline and from Day 0 to Day 360
Number of Participants With Adverse Events of Special Interest as a Measure of Safety and Tolerability Based on Dose
时间窗: Up to Day 390
Columbia Suicide Severity Rating Scale (C-SSRS), Categorisation Based on Columbia Classification Algorithm of Suicide Assessment (C-CASA) Categories (1, 2, 3, 4 and 7) for Patients Aged ≥ 6 Years
时间窗: Baseline and from Day 0 to Day 360
次要结局
- Change in Mean Weekly Number of Tonic-clonic and Clonic Seizures(Baseline and from Day 0 to Day 360 and upon Study Completion/Withdrawal)
- Percentage of Initial Treatment Responders Who Returned to Their Baseline Tonic-clonic and Clonic Seizure Rate During the Study (an Assessment of Tachyphylaxis)(Baseline and from Day 0 to Day 360)
- Number of Initial Treatment Responders Who Returned to Their Baseline Tonic-clonic and Clonic Seizure Rate During the Study (an Assessment of Tachyphylaxis)(Baseline and from Day 0 to Day 360)