PILOT TRIAL IN YOUNG PATIENTS WITH IGA NEPHROPATHY

此应用程序的目标是描述一项试点研究，以评估 隔日强的松治疗IgA的疗效观察 儿童、青少年和年轻人的肾病(IgAN)。详细信息： 提供了研究设计、管理和统计分析。 虽然IgAN是一种肾脏疾病，在#年首次被描述 1967年法国，目前的治疗方案中没有一种是经过验证的 利益。在过去的几年里，一些小规模的研究表明 强的松是有益的，但这些都不是决定性的。这个 缺乏已证实的治疗IgAN的方法令人担忧，因为现在很明显 未经治疗的IgAN进展为终末期肾病的比例高达50% 是ESRD患者总数的重要贡献者 美国。 拟议的试点研究是一项多中心、双盲、安慰剂- 安慰剂2的有效性和安全性的随机对照临床试验 隔日强的松治疗年数约为90-100 患有中度严重IgAN的儿童、青少年和青壮年。 因为我们已经为患者进入这一领域定义了相当严格的标准 试验，个别肾病中心符合条件的患者数量 都会很小。因此，将有大量的中心(34) 参与，以招募足够的有效患者 统计分析。为了顺利完成这项研究， 必须认真协调34个中心的活动。 这将通过西南行政中心实现 儿科肾病研究小组(SPNSG)，一个合作小组，有 14年来，我一直在研究IgAN的各个方面。来处理 这项多中心临床试验的复杂性，我们将建立 监控用药和患者的严格程序 合规性、数据管理和分析、临床和实验室 测量，并将与34个 参赛中心。这项为期3年的试验性研究的结果将是 由经验丰富的数据和统计核心的成员分析 阿拉巴马州伯明翰。从这些试点数据中，我们将确定是否完全- 规模化临床试验是合理可行的。如果是这样的话，一个 竞争性续签申请将提交给美国国立卫生研究院。

The objective of this application is to describe a pilot study to evaluate the efficacy of alternate-day prednisone in the treatment of IgA nephropathy (IgAN) in children, adolescents, and young adults. Details of the study design, administration, and statistical analysis are provided. Although IgAN is a disorder of the kidneys that was first described in France in 1967, none of the current treatment options are of proven benefit. Over the past few years, a number of small studies have suggested that prednisone is beneficial, but none of these have been definitive. The lack of proven therapy for IgAN is of concern because it is now apparent that untreated IgAN progresses to end-stage renal disease in up to 50% of patients and is a significant contributor to the total ESRD population in the United States. The proposed pilot study is a multicenter, double-blind, placebo- controlled, randomized clinical trial of the efficacy and safety of 2 years of therapy with alternate-day prednisone in approximately 90-100 children, adolescents, and young adults with moderately severe IgAN. Since we have defined rather strict criteria for patient entry into this trial, the number of eligible patients in individual nephrology centers will be small. Hence, a large number of centers (34) will be participating in order to recruit sufficient patients for valid statistical analysis. In order to successfully complete this study, it is essential that the activities of the 34 centers are carefully coordinated. This will be achieved through the Administration Center of the Southwest Pediatric Nephrology Study Group (SPNSG), a collaborative group that has been studying various aspects of IgAN for 14 years. To handle the complexities of this multicenter, clinical trial, we will establish rigorous procedures for monitoring medication administration and patient compliance, data management and analysis, clinical and laboratory measurements, and will maintain close communication with the 34 participating centers. Results from this 3-year pilot study will be analyzed by members of an experienced Data and Statistics Core in Birmingham, Alabama. From these pilot data, we will determine if a full- scale clinical trial is justified and feasible. if this is the case, a competitive renewal application will be submitted to the NIH.