A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 3
- 主要终点
- Electroclinical response (Efficacy)
研究概览
简要总结
This is a phase II clinical trial in which children with refractory infantile spasms (also called epileptic spasms or West syndrome) will be treated with fenfluramine, to evaluate efficacy, safety, and tolerability. Patients with infantile spasms that have not responded to treatment with vigabatrin and ACTH we will be invited to participate. Study participants will undergo baseline video-EEG, receive treatment with fenfluramine for 21 days, and then undergo repeat video-EEG to determine effectiveness. Patients with favorable response will have the opportunity to continue treatment for up to 6 months.
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
- 12 Months 至 36 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children ages 12 to 36 months, inclusive
- •Clinical diagnosis of infantile spasms
- •Continued epileptic spasms despite adequate treatment with ACTH and vigabatrin.
排除标准
- •Significant preexisting cardiovascular disease
- •Exposure to any cannabinoid product within 14 days of screening
- •Initiation or dose-titration of any second-line treatment for infantile spasms in the 14 days prior to screening.
- •Implantation of a vagal nerve simulator within 14 days of screening
- •Initiation and maintenance of the ketogenic diet within 3 months of screening
研究组 & 干预措施
Fenfluramine treatment
Open label treatment with fenfluramine. Dosage will be titrated to 0.8 mg/kg/day, for an initial duration of 21 days. Patients with favorable response will have an option to continue treatment for up to 6 months.
干预措施: Fenfluramine (Drug)
结局指标
主要结局
Electroclinical response (Efficacy)
时间窗: 12 months
Number of participants with resolution of epileptic spasms and hypsarrhythmia (if present at baseline) after 21 days of treatment, as determined by overnight video-electroencephalography (EEG) evaluation and caregiver seizure diary.
次要结局
- Incidence of treatment emergent adverse events (Safety and tolerability)(12 months)
- Computational electroencephalography response (Efficacy)(12 months)
研究者
Daniel Shrey
Associate Professor
Children's Hospital of Orange County