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

DMD 皮质类固醇治疗方案试验的生物统计学和数据管理

• 批准号: 9763665
• 负责人: Michael P McDermott
• 金额: $ 20.4万
• 依托单位: UNIVERSITY OF ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-07-01 至 2021-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9763665
• 关键词: 7 year old; Address; Adrenal Cortex Hormones; Adverse event; Age; Biometry; Child; Clinic; Clinical Trials Data Monitoring Committees; Companions; Computational Biology; Country; Data; Data Reporting; Databases; Dimensions; Disclosure; Doctor of Medicine; Double-Blind Method; Duchenne muscular dystrophy; Emergency treatment; Enrollment; Environment; Event; Funding; Future; Generations; Goals; Heart Research; Heterogeneity; Human Resources; Infrastructure; International; Internet; Maintenance; Measures; Medical center; Modification; Monitor; Multi-Institutional Clinical Trial; Muscle; National Institute of Neurological Disorders and Stroke; Neurology; Neuromuscular Diseases; Online Systems; Outcome; Parents; Participant; Patients; Pharmaceutical Preparations; Prednisone; Preparation; Procedures; Questionnaires; Randomized; Regimen; Reporting; Request for Applications; Resolution; Risk; Safety; Sample Size; Secure; Serious Adverse Event; Site; Standardization; Statistical Data Interpretation; Steroids; Suspensions; System; Testing; Time; Training; Treatment Protocols; Universities; Visit; Vital capacity; Withdrawal; base; clinical practice; clinically relevant; clinically significant; data management; deflazacort; design; dosage; evidence base; follow-up; premature; primary outcome; programs; randomized trial; recruit; relational database; satisfaction; screening; treatment group; trial comparing

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.

该申请要求继续为数据管理和生物统计支持提供资金 国际多中心临床试验比较三种不同的皮质类固醇 (CS) 治疗方案 杜氏肌营养不良症 (DMD)。这是凯特布什比医学博士的配套应用程序 纽卡斯尔大学（英国）和罗彻斯特大学（美国）医学博士 Robert C. Griggs 题为 “FOR-DMD：优化 Duchenne MD 类固醇治疗方案的双盲随机试验。”初级 该项目的目的是进行一项国际多中心、随机、双盲、对照试验，其中 225 名 4 岁至 7 岁之间患有 DMD 的儿童将被随机分组​​接受每日泼尼松（0.75 mg/kg），每日地夫可特（0.90 mg/kg），或间歇性（10天，停10天）泼尼松（0.75 mg/kg）和 跟踪至少三年。主要结果变量将是三维的，包括 (1) 从躺着起立的时间（对数转换），(2) 用力肺活量，以及 (3) 受试者/家长的全局 对治疗的满意度，是三年随访期间所有基线后就诊的平均值。 该试验将解决一个务实的假设，即每日泼尼松和每日地夫可特会产生更大的效果。 与在一段时间内给予间歇性 CS（泼尼松）相比，在功能和受试者/家长满意度方面的益处 三年期限。目前，已有 174 名参与者（77%）参加了该试验，预计招募人数将在 2016 年 9 月完成。 我们应用程序的具体目标是（1）为试验提供数据管理，包括：实施 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到一个 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 随机化和数据输入、数据查询的生成和解决、重要研究事件的监测、 维护适当安全且合规的网络/数据库环境； (2) 至 为试验提供生物统计支持，包括： 统计分析的完善和实施 计划解决试验的具体目标；实施临时监测计划，使 根据安全问题的早期证据修改或终止研究，并结合 NINDS 指定的数据和安全监测委员会 (DSMB)；以及公开会议和闭门会议的准备 DSMB 的报告。