Established Status Epilepticus Treatment Trial (ESETT)

已建立的癫痫持续状态治疗试验 (ESETT)

• 批准号: 9234074
• 负责人: James Chamberlain
• 金额: $ 350.43万
• 依托单位: UNIVERSITY OF VIRGINIA
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-30 至 2020-02-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9234074
• 关键词: 2 year old; Accident and Emergency department; Admission activity; Adult; Adverse effects; Adverse reactions; Affect; Age; Anticonvulsants; Applied Research; Arrhythmia; Benzodiazepines; Blinded; Brain Injuries; Cessation of life; Child; Childhood; Clinical; Clinical Research; Clinical Trials; Clinical Trials Design; Conscious; Diazepam; Dose; Double-Blind Method; Double-blind trial; Effectiveness; Eligibility Determination; Emergency Care; Emergency Situation; Emergency research; Emergency treatment; Enrollment; Ensure; Etiology; Fever; Futility; Goals; Hour; Hypotension; Individual; Informed Consent; Infusion procedures; Intervention; Intravenous; Intubation; Levetiracetam; Life; Lorazepam; Nervous System Trauma; Neurological emergencies; Neurology; Outcome Measure; Patient Recruitments; Patients; Pharmaceutical Preparations; Phase; Phase III Clinical Trials; Probability; Professional Organizations; Protocols documentation; Randomized; Randomized Controlled Clinical Trials; Recovery; Recruitment Activity; Recurrence; Refractory; Regulation; Research; Research Personnel; Resolution; Risk; Sample Size; Seizures; Serious Adverse Event; Status Epilepticus; Testing; Time; Tonic-Clonic Epilepsy; Valproic Acid; Vial device; Weight; Work; adverse outcome; age group; arm; base; comparative effectiveness; comparative efficacy; data management; design; effective therapy; effectiveness measure; effectiveness trial; experience; fosphenytoin; improved; mental state; mortality; older patient; primary outcome; prospective; public health relevance; randomized trial; response; secondary outcome; success; treatment group; treatment trial

DESCRIPTION (provided by applicant): Benzodiazepine-refractory status epilepticus (Established Status Epilepticus, ESE) is a relatively common emergency condition with several widely used treatments. There are no controlled, randomized, blinded clinical trials to compare the efficacy and tolerability of currently available treatments of ESE. This and the accompanying Statistical and Data Management Center (SDMC) application describe the ESE treatment trial (ESETT), which is designed to determine the most effective and/or the least effective treatment of ESE among patients older than two years by comparing three arms: fosphenytoin (FOS), levetiracetam (LEV), and valproic acid (VPA). This is a multicenter, randomized, double-blind, Bayesian adaptive, Phase III comparative effectiveness trial. Up to 795 patients will be randomized initially 1:1:1 and response-adaptive randomization will occur after 300 patients have been recruited. Randomization will be stratified by three age groups, 2-18, 19-65, and 66 years and older. The primary outcome measure is cessation of clinical seizure activity and improving mental status, without serious adverse effects or further intervention at 60 min after administration of study drug. Each subject will be followed until discharge or 30 days from enrollment. This trial will include interim analyses for early success and futility. This trial wil be considered a success if the probability that a treatment is the most effective is greater than 0.975 or the probability that a treatment is the least effective is greater than 0.975 for any treatment. This will be the first phase III clinical trial of ESE in children and adults.

描述（由申请人提供）：苯二氮卓类难治性癫痫持续状态（既定癫痫持续状态，ESE）是一种相对常见的紧急情况，有几种广泛使用的治疗方法。目前尚无对照、随机、盲法临床试验来比较目前可用的 ESE 治疗方法的疗效和耐受性。本申请以及随附的统计和数据管理中心 (SDMC) 申请描述了 ESE 治疗试验 (ESETT)，该试验旨在通过比较三个组来确定两岁以上患者中最有效和/或最无效的 ESE 治疗：磷苯妥英 (FOS)、左乙拉西坦 (LEV) 和丙戊酸 (VPA)。这是一项多中心、随机、双盲、贝叶斯自适应、III 期比较有效性试验。最初将按照 1:1:1 的比例对最多 795 名患者进行随机分组，并在招募 300 名患者后进行反应适应性随机分组。随机分组将按三个年龄组进行分层：2-18 岁、19-65 岁和 66 岁及以上。主要结局指标是在给予研究药物 60 分钟后临床癫痫活动停止并改善精神状态，没有严重的不良反应或进一步干预。每个科目都将被跟踪直至出院或入组后 30 天。该试验将包括对早期成功和无效的中期分析。对于任何治疗，如果治疗最有效的概率大于 0.975 或治疗最不有效的概率大于 0.975，则该试验将被视为成功。这将是 ESE 在儿童和成人中的第一个 III 期临床试验。