Biostatistics and Data Management for a Trial of Corticosteroid Regimens in DMD

该应用程序要求继续为数据管理和生物统计支持提供资金 三种不同的皮质类固醇(CS)方案治疗卵巢癌的国际多中心临床试验 Duchenne肌营养不良症(DMD)。这是凯特·布什比医学博士的一个配套应用程序 纽卡斯尔大学(英国)和罗切斯特大学(美国)Robert C.Griggs医学博士，题为 FOR-DMD：双盲随机试验，以优化Duchenne MD的类固醇治疗方案。初级阶段 该项目的目标是进行一项国际多中心、随机、双盲、对照试验 225名4岁至7岁的DMD儿童将随机接受每日泼尼松(0.75)治疗 每日地卡西林(0.90 mg/kg)，或间歇性(服药10天，停药10天)强的松(0.75 mg/kg)和 至少跟踪了三年。主要结果变量将是三维的，并将包括 (1)从卧床站立的时间(对数变换)，(2)用力肺活量，以及(3)受试者/父母全局 对治疗的满意度，在三年的随访期内，所有患者在基线后的所有就诊中平均得分。 这项试验将解决以下实用假设：每日强的松和每日呋喃西林的疗效将更好 在功能和受试者/父母满意度方面的好处比间断服用CS(泼尼松)要好 三年期。目前，174名参与者(77%)参加了试验，预计将招募 于2016年9月完成。 我们的应用程序的具体目标是(1)为试验提供数据管理，包括：实现 基于网络的随机化和登记程序，通过互联网将所有研究数据输入 标准化关系数据库，培训现场人员使用基于网络的应用程序 随机化和数据录入、数据查询的生成和解决、重要研究事件的监控， 以及维持适当安全和合规的网络/数据库环境；及 为试验提供生物统计支持，包括：改进和实施统计分析 计划实现审判的具体目标；执行临时监测计划，以使 根据安全问题的早期证据修改或终止研究，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 continued funding for the data management and biostatistical support for an international multicenter clinical trial comparing three different corticosteroid (CS) regimens for the treatment of Duchenne muscular dystrophy (DMD). This is a companion application to that of Kate Bushby, M.D. from the University of Newcastle (UK) and Robert C. Griggs, M.D. from the University of Rochester (USA) entitled “FOR-DMD: Double-Blind Randomized Trial to Optimize Steroid Regimens in Duchenne MD.” The primary aim of this project is to conduct an international multicenter, randomized, double-blind, controlled trial in which 225 children with DMD between the ages of 4 and 7 will be randomized to receive either daily prednisone (0.75 mg/kg), daily deflazacort (0.90 mg/kg), or intermittent (10 days on, 10 days off) prednisone (0.75 mg/kg) and followed for a minimum of three years. The primary outcome variable will be three-dimensional and will consist of (1) time to stand from lying (log-transformed), (2) forced vital capacity, and (3) subject/parent global satisfaction with treatment, all averaged over all post-baseline visits during the three-year follow-up period. The trial will address the pragmatic hypothesis that daily prednisone and daily deflazacort will be of greater benefit in terms of function and subject/parent satisfaction than intermittent CS (prednisone) given over a three-year period. Currently, 174 participants (77%) are enrolled in the trial and recruitment is expected to be completed by September 2016. The specific aims of our application are (1) To provide data management for the trial including: implementation of web-based randomization and enrollment procedures, internet-facilitated entry of all study data into a standardized relational database, training of site personnel in the use of web-based applications for randomization and data entry, generation and resolution of data queries, monitoring of important study events, and maintenance of an appropriately secured and compliant network/database environment; and (2) To provide biostatistical support for the trial including: refinement and implementation of the statistical analysis plan to address the specific aims of the trial; implementation of an interim monitoring plan that will allow modification or termination of the study on the basis of early evidence of safety concerns, in conjunction with the NINDS-appointed Data and Safety Monitoring Board (DSMB); and preparation of open- and closed-session reports for the DSMB.