A Multicenter Pilot Clinical Trial to Prevent Infantile Spasms Relapse
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Incidence of treatment-emergent adverse events
研究概览
简要总结
After initially successful treatment, many children with infantile spasms unfortunately have a relapse, and relapse is linked to poor long-term outcomes such as autism and other forms of epilepsy. The aim of this study is to determine if treatment with low-dose prednisolone is safe, well tolerated, and effective in reducing the risk of relapse.
详细描述
In this study, the investigators will enroll infants who have responded to standard therapy for treatment of Infantile Epileptic Spasms Syndrome (IESS). Patients will be randomized to receive either low-dose prednsiolone or placebo for 4 months. During the first 7 months, patients will be evaluated monthly, with clinic visits and electroencephalography (EEG). Patients will then return for a final visit at age 2 years. Key outcomes will be determination of IESS relapse, emergence of other types of seizures/epilepsy, and evaluation of developmental/behavioral status at age 2 years. We will attempt to determine whether or not the prednisolone intervention reduces the risk of any adverse outcome, but this may not be possible given the study design. We will also evaluate the feasibility of the intervention, study procedures, and recruitment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Factorial
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Months 至 18 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 2 to 18 months, inclusive
- •Clinical diagnosis of infantile spasms syndrome, with EEG-confirmed complete response to standard treatment (prednisolone, ACTH, and/or vigabatrin)
排除标准
- •Presence of clinically significant hypertension, infection, or any other diagnosis which poses unreasonable risk in the setting of extended corticosteroid therapy, in the view of the study physician
- •Exposure to any artisanal cannabinoid product within 14 days of screening
- •Ongoing therapy with the ketogenic diet
- •Implantation of a vagal nerve stimulator within 3 months of screening, or any change in stimulation parameters within 1 month of screening
- •Treatment of IESS via epilepsy surgery
研究组 & 干预措施
Low-dose prednisolone
Prednisolone and famotidine.
干预措施: Prednisolone (Drug)
Low-dose prednisolone
Prednisolone and famotidine.
干预措施: Famotidine (Drug)
Placebo
Placebo (prednisolone) and placebo (famotidine)
干预措施: Placebo (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events
时间窗: From enrollment to 5-month visit.
The investigators will tabulate of adverse events and determine whether any adverse event is associated with prednisolone treatment during the first 5 months of the study.
Incidence of epileptic spasms relapse
时间窗: From enrollment to last evaluation at age 2 years
The investigators will record whether or not study participant experience a relapse of epileptic spasms during the study. Relapse is classified as present or absent and based on interpretation of EEG.
次要结局
- Incidence of autism spectrum disorder(From enrollment to last evaluation at age 2 years)
- Developmental/Behavioral Level(From enrollment to last evaluation at age 2 years)
研究者
Shaun Hussain, MD
Professor
University of California, Los Angeles