Comparison of Cyclosporine A and Mycophenolate in FSGS

描述（由申请人提供）： 本申请是根据NIH-RFA-DK-02-013提交的。本申请的目的是申请西南儿科肾病组（SPNSG）中心办公室被选为全国儿童和年轻成人肾病综合征和局灶节段性肾小球硬化症（FSGS）治疗方案随机临床试验（RCT）的四个区域中心之一。SPNSG负责协调儿童肾病的多中心临床试验已有二十（20）年，过去六（6）年负责协调成人伊加肾病的多中心临床试验。我们目前正在进行一项多中心试验，评价吗替麦考酚酯治疗儿童类固醇依赖性肾病综合征的疗效，我们正在开始一项新的随机对照试验，用于7至70岁的伊加肾病患者。我们中心的位置和我们在开发多中心研究网络方面的经验，将使我们成为一个非常有效的研究中心，为美国西南部和中西部地区的患者和医生开展研究。该提案的具体内容为正在设计的RCT提供了一种方法，并根据RFA的要求，提供了有关我们招募患者和中心方法的详细信息。我们计划覆盖的美国特定区域包括十四（14）个州的主要肾病中心：德克萨斯州、路易斯安那州、俄克拉荷马州、阿肯色州、堪萨斯、田纳西州、密西西比、密苏里州、科罗拉多、亚利桑那、伊利诺伊、犹他州、新墨西哥州和肯塔基州。 我们提出的试验包括一个安慰剂对照的评估两种免疫调节药物（环孢素A和霉酚酸酯）治疗FSGS在一个大的儿童和成人年龄小于40岁。所有患者（包括“安慰剂”组）将接受血管紧张素转换酶抑制剂（依那普利）-我们认为这是标准治疗护理。患者将接受2年的治疗，在此期间，我们将测量他们的尿蛋白水平作为肾脏疾病的标志物。2年疗程完成后，这些测量将再持续六个月。我们提出的治疗方法的最终目标是使患者能够保留其肾功能，避免后续肾移植的需要。

DESCRIPTION (provided by applicant): This application is being submitted in response to NIH-RFA-DK-02-013. The purpose of the application is to apply for the Central Office of the Southwest Pediatric Nephrology Group (SPNSG) to be chosen as one of the four Regional Centers in the nationwide randomized clinical trial (RCT) of treatment options for children and young adults with nephrotic syndrome and focal segmental glomerulosclerosis (FSGS). The SPNSG has been responsible for coordinating multicenter clinical trials in children with kidney disease for twenty (20) years, and for the past six (6) years in adults with IgA Nephropathy. We are currently conducting a multicenter trial evaluating the efficacy of mycophenolate mofetil in children with steroid dependent nephrotic syndrome and we are starting a new RCT in patients with IgA Nephropathy who are aged 7 to 70 years of age. The location of our center and the experience we have had in developing networks for multicenter studies, will allow us to be a very effective site for conducting the study for patients and physicians in the Southwest and Midwest regions of the United States. The specifics of the proposal provide one approach to the RCT that is being designed and, as requested in the RFA, provides details regarding our approach to patient and center recruitment. The specific region of the U.S.A. that we propose to cover encompasses the major Nephrology Centers in fourteen (14) states: Texas, Louisiana, Oklahoma, Arkansas, Kansas, Tennessee, Mississippi, Missouri, Colorado, Arizona, Illinois, Utah, New Mexico and Kentucky. The trial that we are proposing involves a placebo-controlled evaluation of two immunomodulating drugs (cyclosporine A and mycophenolate mofetil) for the treatment of FSGS in a large group of children and adults less than 40 years of age. All of the patients (including the "placebo" group) will receive an angiotensin converting enzyme inhibitor (enalapril)-which we consider to be standard care of therapy. The patients will be treated for 2 years during which we will be measuring their urinary protein levels as a marker of kidney disease. These measurements will be continued for an additional six months after the 2-year course of therapy has been completed. Our ultimate goal with the treatments proposed is to enable the patients to preserve their kidney function and avoid the need for subsequent kidney transplantation.