Childhood Absence Epilepsy: Coordinating Center

儿童失神癫痫：协调中心

基本信息

批准号：
7340707
负责人：
AVITAL CNAAN
金额：
$ 76.03万
依托单位：
CHILDREN'S HOSP OF PHILADELPHIA
依托单位国家：
美国
项目类别：
财政年份：
2003
资助国家：
美国
起止时间：
2003-09-30 至 2008-10-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7340707
关键词：
8 year old Absence Epilepsy Address Adherence Adverse effects Affect Age Anticonvulsants Antiepileptic Agents Arts Behavioral Bilateral Biological Markers Biometry Case Report Form Characteristics Child Childhood Clinical Clinical Research Clinical Trials Clinical Trials Data Monitoring Committees Communication Companions Complex Computers Conscious Consensus Controlled Clinical Trials Coupled Data Data Analyses Data Collection Data Quality Databases Development Disease Disease remission Dose Dose-Limiting Double-Blind Method Drug Exposure Drug Kinetics Effectiveness Electroencephalography Eligibility Determination End Point Enrollment Ensure Environment Epilepsy Ethosuximide Failure Freedom Frequencies Future Genetic Genotype Goals Guidelines Heterogeneity Human Resources Imaging problem Impairment Incidence Individual Intelligence Lead Leadership Link Manuals Manuscripts Medical Monitor Nature Operative Surgical Procedures Patients Pattern Pediatric Hospitals Pediatric Neurology Performance Pharmaceutical Preparations Pharmacogenetics Pharmacotherapy Pharmacy facility Phenotype Philadelphia Population Population Heterogeneity Preparation Procedures Protocols documentation Publishing Quality Control Randomized Range Rate Reader Regulation Reporting Research Research Personnel Resources Safety Screening procedure Secure Seizures Self Perception Site Standards of Weights and Measures Syndrome Teleconferences Therapeutic Time Today Toxic effect Training Transference Treatment Failure United States United States National Institutes of Health Valproic Acid Variant Work age related base clinical phenotype data integrity data management design experience lamotrigine multidisciplinary organizational structure performance site preference programs prospective repository response success

项目摘要

DESCRIPTION (provided by the applicant): Treatment for Childhood Absence Epilepsy (CAE) is largely empiric. Although there are three efficacious antiepileptic drugs (AEDs) - ethosuximide (ETX), lamotrigine (LTG), valproic acid (VPA) - each has variable success in seizure control, as well as treatment-limiting toxicities. This project is being resubmitted as two linked R01 applications. The clinical proposal, "Childhood Absence Epilepsy - Rx, PK/PK, Genetics," (PI: T. Glauser, Co-PI: P. Adamson) is being resubmitted by the Cincinnati Children's Hospital. The objectives of the clinical trial are twofold: a) identify the optimal initial AED in terms of seizure control and lowest toxicity incidence; and b) to determine the clinical, pharmacokinetic and pharmacogenetic factors underlying the inter-individual variation in AED response and toxicity. This linked R01 proposal will establish the Childhood Absence Epilepsy Coordinating Center [CHAECC] at The Children's Hospital of Philadelphia to support this broad, integrated plan to study CAE. A strong and efficient coordinating center is needed in a project of this magnitude and complexity to ensure that the scientific goals are achieved in a timely manner and to a high standard of scientific excellence and data integrity as well as subject safety. The Children's Hospital of Philadelphia (CHOP) has established a highly experienced team to provide this coordinating center function. The aims of the CHAECC are to provide: 1) operational support to implement the protocol; 2) data base and data management for the protocol and the Core's data; 3) biostatistical analyses of the project aims. To meet these goals, the CHAECC will perform start-up activities to implement the project, coordinate all communication activities, oversee adherence to Good Clinical Practice and HIPPA guidelines in protocol implementation, and will work closely with the Scientific Leadership of the study. The CHAECC will develop procedures for data collection, transference, and storage in a secure relational database. The CHAECC will coordinate receipt of video EEG's by Core readers and meetings to achieve consensus in interpretations. It will conduct all statistical analyses and support preparation of reports, manuscripts and presentations.

描述（由申请人提供）：儿童失神癫痫（CAE）的治疗主要是经验性的。尽管存在三种有效的抗癫痫药物 (AED)——乙舒酰亚胺 (ETX)、拉莫三嗪 (LTG)、丙戊酸 (VPA)，但每种药物在控制癫痫发作方面的成功率各不相同，并且存在限制治疗的毒性。该项目将作为两个链接的 R01 申请重新提交。辛辛那提儿童医院正在重新提交临床提案“儿童失神癫痫 - Rx、PK/PK、遗传学”（PI：T. Glauser，Co-PI：P. Adamson）。临床试验的目标有两个： a) 确定在癫痫发作控制和最低毒性发生率方面的最佳初始 AED； b) 确定 AED 反应和毒性个体间差异背后的临床、药代动力学和药物遗传学因素。这项相关的 R01 提案将在费城儿童医院建立儿童失神癫痫协调中心 [CHAECC]，以支持这项广泛、综合的 CAE 研究计划。如此规模和复杂的项目需要一个强大而高效的协调中心，以确保及时实现科学目标，并达到高标准的科学卓越性、数据完整性以及受试者安全性。费城儿童医院 (CHOP) 建立了一支经验丰富的团队来提供这一协调中心职能。 CHAECC 的目标是提供： 1）实施协议的操作支持； 2）协议和核心数据的数据库和数据管理； 3）项目目标的生物统计分析。为了实现这些目标，CHAECC 将开展启动活动来实施该项目，协调所有沟通活动，监督方案实施中对良好临床实践和 HIPPA 指南的遵守情况，并将与该研究的科学领导层密切合作。 CHAECC 将开发数据收集、传输和存储在安全关系数据库中的程序。 CHAECC 将协调核心读者对视频脑电图的接收和会议，以在解释上达成共识。它将进行所有统计分析并支持报告、手稿和演示文稿的准备。

项目成果

期刊论文数量（2）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

Pretreatment behavior and subsequent medication effects in childhood absence epilepsy.

儿童失神癫痫的治疗前行为和随后的药物作用。

DOI：
10.1212/wnl.0000000000004514
发表时间：
2017
期刊：
Neurology
影响因子：
9.9
作者：
Shinnar,RuthC;Shinnar,Shlomo;Cnaan,Avital;Clark,Peggy;Dlugos,Dennis;Hirtz,DeborahG;Hu,Fengming;Liu,Chunyan;Masur,David;Weiss,EricaF;Glauser,TracyA;ChildhoodAbsenceEpilepsyStudyGroup
通讯作者：
ChildhoodAbsenceEpilepsyStudyGroup