Coordination and Statistics for CoQ10 in HD [2CARE]

该应用程序请求为研究协调、数据管理和生物统计学支持提供资金 辅酶Qio(CoQ)治疗亨廷顿病(HD)的第三阶段临床试验。这是一位同伴 对马萨诸塞州总医院医学博士Merit E.Cudkowicz的应用 亨廷顿病中的QIO(2CARE)。该项目的主要目标是进行一个多中心 每天2400毫克辅酶Q的随机、双盲、安慰剂对照试验，涉及1100人 患有HD的非卧床受试者前瞻性地随访了五年。辅酶Q是一种辅助因子，参与 线粒体电子转移和一种抗氧化剂，已证明提高了转基因动物的存活率 HD的小鼠模型，以及在HD和帕金森病的人体试验中的有希望的结果。这是假设的 大剂量辅酶Q的慢性治疗将减缓HD的进行性功能衰退。 我们申请的具体目标是(1)为试验提供项目协调，包括： 制定和实施基于网络的随机化和注册程序，提供研究 文件(协议、示范同意书和同意表、操作手册、案例报告表)、现场培训 人员和学习程序的标准化，便利招聘和留住多样化的 研究对象的抽样，研究药品的包装和分发；(2)提供数据管理 试验包括：接收和输入关系数据库中的所有数据，生成和解析数据 查询、监测重要研究事件以及维护合规和安全的数据环境； 以及(3)为试验提供生物统计学支持，包括：完善和实施 统计分析计划，以解决试验的具体目标；设计和实施临时 监测计划，将允许根据以下早期证据修改或终止研究 (I)安全问题，(Ii)压倒性的有效性证据，或(Iii)无效，与 NINDS任命的数据和安全监测委员会(数据和安全监测委员会)；筹备开幕和闭门会议 针对DSMB的报告。

This application requests funding for the study coordination, data management, and biostatistical support for a Phase III clinical trial of Coenzyme Qio (CoQ) in Huntington's disease (HD). This is a companion application to that of Merit E. Cudkowicz, M.D. from Massachusetts General Hospital entitled "Coenzyme Qio in Huntington's Disease (2CARE)." The primary aim of the project is to conduct a multicenter randomized, double-blind, placebo-controlled trial of CoQ at a dosage of 2400 mg/day involving 1100 ambulatory subjects with HD followed prospectively for five years. CoQ is a cofactor involved in mitochondrial electron transfer and an antioxidant that has demonstrated increased survival in a transgenic mouse model of HD and promising results in human trials in HD and Parkinson's disease. It is hypothesized that chronic treatment with high-dosage CoQ will slow the progressive functional decline in HD. The specific aims of our application are (1) To provide project coordination for the trial including: development and implementation of web-based randomization and enrollment procedures, provision of study documents (protocol, model consent and assent forms, operations manual, case report forms), training of site personnel and standardization of study procedures, facilitation of recruitment and retention of a diverse sample of research subjects, and packaging and distribution of study drug; (2) To provide data management for the trial including:receipt and entry of all data in a relational database, generation and resolution of data queries, monitoring of important study events, and maintenance of a compliant and secure data environment; and (3) To provide biostatistical support for the trial including: refinement and implementation of the statistical analysis plan to address the specific aims of the trial; design and implementation of an interim monitoring plan that will allow modification or termination of the study on the basis of early evidence of either (i) safety concerns, (ii) overwhelming evidence of efficacy, or (iii) futility, in conjunction with the NINDS-appointed Data and Safety Monitoring Board (DSMB); and preparation of open- and closed-session reports for the DSMB.