PILOT TRIAL IN YOUNG PATIENTS WITH IGA NEPHROPATHY

本申请的目的是描述一项初步研究，以评估 泼尼松隔日疗法治疗IgA肾病的疗效观察 肾病（IgAN）在儿童，青少年和年轻人。详情 提供了研究设计、管理和统计分析。 虽然IgAN是一种肾脏疾病， 法国在1967年，目前的治疗方案都没有得到证实 效益在过去的几年里，一些小型研究表明， 强的松是有益的，但这些都不是确定的。的 缺乏已证实的IgAN治疗是令人关注的，因为现在很明显 未经治疗的IgAN在高达50%的人中进展为终末期肾病， 是总ESRD人群的重要贡献者， 美国的 拟议的初步研究是一项多中心、双盲、安慰剂- 对照，随机临床试验的有效性和安全性2 在大约90 - 100年中， 中重度IgAN的儿童、青少年和年轻成人。 由于我们已经为患者进入这个领域制定了相当严格的标准， 试验中，各个肾脏病中心合格患者的数量 会很小。 因此，将有大量中心（34个） 参与，以招募足够的患者， 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， 研究IgAN的各个方面已经14年了。办理 这项多中心临床试验的复杂性，我们将建立 严格的程序来监测药物管理和患者 合规性、数据管理和分析、临床和实验室 并将与34个国家保持密切联系。 参与中心。 这项为期3年的试点研究的结果将是 由经验丰富的数据和统计核心的成员分析， 伯明翰，亚拉巴马。从这些试验数据中，我们将确定是否全面- 大规模临床试验是合理可行的。如果是这种情况， 竞争性更新申请将提交给NIH。

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.