NCT00442104终止2 期
An Open-label Clinical Study to Evaluate the Safety and Antiepileptic Activity of Ganaxolone in Treatment of Patients Diagnosed With Infantile Spasms.
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 54
- 试验地点
- 14
- 主要终点
- Percentage of Participants Who Were Free of Spasms
研究概览
简要总结
To allow open-label extension to patients who have completed Protocol 1042-0500
详细描述
Patient should have completed all scheduled clinical study visits in the double blind, controlled trial (Protocol 1042-0500) and have been deemed eligible (had a response to treatment) by the Investigator. Male or female, with a diagnosis of IS with a video EEG (vEEG) recording confirming the diagnosis.
There will be a total of 14 visits over 99(+or-1)week. A 24-hr vEEG is only required if the subject has been spasm-free for more than 24-hrs.
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
- 4 Months 至 24 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have completed all scheduled clinical study visits in the previous Protocol 1042 0500 and have been deemed eligible (no SAEs thought to be drug related and had a response to treatment) by the Investigator.
- •Be diagnosed with IS regardless of etiology. Diagnostic Criteria: Seizures consisting of single or repetitive short muscular contractions leading to flexion or extension. Spasms may be characterized as tonic or myoclonic contractions, may occur singly or in clusters, and typically occur bilaterally and symmetrically. The EEG pattern must be consistent with the diagnosis of IS (hypsarrhythmia, modified hypsarrhythmia, multifocal spike wave discharges, etc).
- •Have a 24 hour vEEG recording confirming the diagnosis of IS.
- •Have had a magnetic resonance imaging (MRI) performed to determine any possible causes of IS.
- •Have been previously treated with 3 AEDs or fewer.
- •Have a parent/guardian who is properly informed of the nature and potential risks and benefits of the clinical study, is willing and capable of complying with all clinical study procedures, and has given informed consent in writing prior to entering the clinical study.
排除标准
- •Current treatment with more than 2 concomitant AEDs.
- •Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive (with the exception of tuberous sclerosis) as evaluated by brain MRI.
- •Have any disease or condition (medical or surgical) at Screening that might compromise the hematologic, cardiovascular, pulmonary, renal, GI, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the investigational product, or would place the subject at increased risk.
- •Aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin greater than 4 times the upper limit of laboratory normal or any clinical laboratory value deemed clinically significant by the Investigator.
- •History of recurrent status epilepticus.
- •Have been exposed to any other investigational drug within 30 days prior to enrollment.
研究组 & 干预措施
ganaxolone
Experimental
干预措施: Ganaxolone (Drug)
结局指标
主要结局
Percentage of Participants Who Were Free of Spasms
时间窗: Weeks 4 through Week 96
Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented.
次要结局
- Change From Baseline in Frequency of Individual Spasm(Baseline and Week 4 through Week 32)
- Number of Participants With Spasm-free Durations(Week 4 through Week 32)
- Change From Baseline in Frequency of Spasm Clusters(Baseline and Week 4 through Week 32)
- Number of Participants With Change in Clinical Status on Caregiver's Global Assessment(Week 4 through Week 32)
- Number of Participants With Change in Clinical Status on the Investigator's Global Assessment(Week 4 through Week 32)
- Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary.(Weeks 4 through Week 32)
- Number of Participants With Absence of Spasms(Week 4 through Week 32)
- Developmental Assessment Using Denver-II Developmental Test(Week 8 through Week 32)
研究者
研究点 (14)
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