Early Aggressive Treatment of Polyarticular JRA

描述(由申请人提供)： 这是一个由两部分组成的应用程序。第1部分：一种简化的随机化方案 多发性JRA早期侵袭性治疗的积极对照试验。第二部分：A 为审判的进行制定必要组成部分的计划。 第1部分：这项试验的总体目标是改善身体机能 多发性硬化症儿童的能力与健康相关生活质量 关节幼年类风湿性关节炎(PolyJRA)。具体目标：比较 一种积极的治疗方案的有效性和安全性 多发性JRA儿童疾病的前6个月到目前的情况 在产生完全临床应答和改善状态方面的护理标准 结果通过为期6个月的随机开放试验。基本假设 是：1)在多发性JRA病后6个月内开始积极治疗 发病将导致更高比例的儿童进入 与完全临床应答的儿童比例进行比较 通过在6个月后使用目前的护理标准来实现这样的反应 接受治疗的机会。2)早期积极的治疗会带来更好的结果 身体/功能能力与目前的护理标准相比 治疗6个月。3)早期积极治疗将导致更高的 与健康相关的生活质量与当前 治疗6个月后进行标准护理。 第2部分：总体目标是为执行 第1部分中描述的临床试验，并为努力获得资金 允许开始审判。具体目标1：通过问卷调查确定 临床医生有效参与临床试验的障碍调查 参与此试验的站点以及其他潜在站点，包括 能够迅速地向患者开放登记，招聘 研究参与者、随访、病例报告表填写。具体目标2： 召集高级调查人员、方法论专家和监管人员召开会议 人员制定战略，以克服 具体目标1.具体目标3：制定界定下列各项之一的标准 主要结果变量，完全临床反应，以及相关术语， 临床缓解，将用于拟议的试验。具体目标4： 准备并提交申请以资助部分所述的临床试验 1.

DESCRIPTION (provided by applicant): This is a 2-part application. PART 1: An abbreviated protocol for a randomized actively controlled trial of early aggressive therapy in poly-JRA. PART II: A plan for development of essential components for the trial's conduct. PART 1: The overall goal of this trial is to improve the physical functional ability and health related quality of life outcomes of children with poly articular juvenile rheumatoid arthritis (poly-JRA). Specific Aim: To compare the efficacy and safety of an aggressive treatment regimen initiated within the first 6 months of disease in children with poly-JRA to that of the current standard-of-care in producing a state of complete clinical response and improve outcome via a randomized, open trial 6 months in duration. Primary hypotheses are: 1) That aggressive therapy initiated within 6-months of poly-JRA disease onset will result in a higher proportion of children entering a state of complete clinical response as compared to the proportion of children who achieve such a response by use of the current standard-of-care after 6 months of therapy. 2) That early aggressive therapy will result in better physical./functional ability as compared to the current standard-of-care after 6 months of therapy. 3) That early aggressive therapy will result in higher health related quality of life as compared to that produced by the current standard-of-care after 6 months of therapy. PART 2: The overall goal is to develop a written plan for the execution of the clinical trial described in PART 1, and to secure funding for the effort to allow commencement of the trial. SpecificAim 1: To determine by questionnaire survey the barriers to the effective participation in trials by the clinical sites involved in this trial as well as other potential sites, including ability to open for patient enrollment in an expeditious manner, recruitment of study participant, follow-up, case report form completion . Specific Aim 2: To convene a meeting of senior investigators, methodologists, and regulatory personnel to develop strategies for overcoming the barriers identified in Specific Aim 1. Specific Aim 3: To develop criteria for defining one of the primary outcome variables, complete clinical response, and the related term, clinical remission, to be used in the proposed trial. Specific Aim 4: To prepare and submit an application to fund the clinical trial described in PART 1.