Multicenter Trial of Focal Glomerulosclerosis

局灶性肾小球硬化症的多中心试验

• 批准号: 6951427
• 负责人: DEBBIE S GIPSON
• 金额: $ 38.86万
• 依托单位: UNIV OF NORTH CAROLINA CHAPEL HILL
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-09-30 至 2007-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6951427
• 关键词: angiotensin receptor; body composition; clinical research; clinical trial phase I; cooperative study; corticosteroids; creatinine; cyclosporines; drug screening /evaluation; epidemiology; glomerulosclerosis; human subject; human therapy evaluation; kidney disorder chemotherapy; kidney pharmacology; losartan; patient oriented research; pharmacokinetics; prednisone; proteinuria; renal failure

DESCRIPTION (provided by applicant): Idiopathic focal segmental glomerulosclerosis (FSGS) is a progressive scarring disorder that causes proteinuria and kidney failure in the majority of affected individuals. There is considerable controversy regarding the best therapeutic intervention and the definition of pathological variants of FSGS that may impact therapeutic response rates. This proposal focuses on the design and conduct of a collaborative multicenter trial that will evaluate response rates of children and young adults with the nephrotic syndrome due to FSGS treated with cyclosporin A as compared to corticosteroids plus angiotensin receptor blocker therapy. It will utilize a newly determined FSGS classification scheme as defined by the NY Pathology Consensus Group that includes one of our collaborators. In addition, since incidence of idiopathic FSGS has been increasing over the past 2 decades, a case-control study that will evaluate risk factors for FSGS is proposed to run concurrently with the trial. Our proposed southeastern clinical coordinating center will provide strength to the planned nation-wide trial through our large patient population with FSGS, the strength of the UNC nephropathology service, and the investigators' long track record of clinical trial and epidemiologic research in glomerular diseases through the UNC-Chapel Hill based Glomerular Disease Collaborative Network. In conjunction with committed collaborating sites, our group has over 400 FSGS patients who would be eligible for the proposed trial, as well as established mechanisms for including prospectively identified patients. The proposed case-control study will evaluate risk factors for the development of FSGS such as body mass, birth weight, viral illnesses and smoking in all patients screened for entry into the trial.

描述（由申请人提供）： 特发性局灶节段性肾小球硬化症 (FSGS) 是一种进行性疤痕性疾病，会导致大多数受影响个体出现蛋白尿和肾衰竭。关于最佳治疗干预和可能影响治疗反应率的 FSGS 病理变异的定义存在相当大的争议。该提案的重点是设计和开展一项协作性多中心试验，该试验将评估因 FSGS 所致肾病综合征的儿童和年轻人接受环孢素 A 治疗与皮质类固醇加血管紧张素受体阻滞剂治疗相比的反应率。它将采用由纽约病理学共识小组定义的新确定的 FSGS 分类方案，其中包括我们的合作者之一。此外，由于特发性 FSGS 的发病率在过去 20 年中一直在增加，因此建议与该试验同时进行一项评估 FSGS 危险因素的病例对照研究。 我们拟建的东南临床协调中心将通过我们庞大的 FSGS 患者群体、北卡罗来纳大学肾病理学服务的实力以及研究人员在肾小球疾病临床试验和流行病学研究方面的长期记录，为计划中的全国性试验提供力量。北卡罗来纳大学教堂山分校肾小球疾病合作网络。与承诺的合作网站相结合，我们的团队拥有超过 400 名 FSGS 患者，他们有资格参加拟议的试验，并建立了纳入前瞻性确定患者的机制。拟议的病例对照研究将评估所有筛选参加试验的患者发生 FSGS 的危险因素，例如体重、出生体重、病毒性疾病和吸烟。