Trial of Cysteamine in Treatment of Corneal Cystine Accumulation in Cystinosis

局部应用半胱胺治疗胱氨酸病中角膜胱氨酸晶体堆积的试验 临床方案支持合同旨在获得开发，设计，解释和评估临床试验，流行病学和自然史研究的校外支持。此外，它将提供涉及眼科疾病和视觉障碍的结果研究以及一些临床前研究。重点应放在研究的设计和收集，分析和解释这些研究产生的数据，以及支持和监测患者的安全性和随访。承包商还应提供工作说明书中所述的分析和数据管理支持，用于指定的临床研究数据库、成本效益和经济分析、生活质量评估和结局研究。这将包括但不限于以下领域：医疗保险和其他医疗保健数据库的分析;现有NEI数据库的评估，如集中的NEI校内研究数据库，眼病病例对照研究，早期治疗糖尿病视网膜病变研究，Fracket Eye研究，以及校内艾滋病和葡萄膜炎数据库。 临床方案的目的： 本研究将继续评价半胱胺滴眼液治疗胱氨酸病患者角膜中胱氨酸晶体的长期安全性和有效性。这些滴剂未上市销售，仅通过本研究提供。新患者可入组研究以获得这些药物。 胱氨酸病是一种遗传性疾病，会导致生长不良和肾脏疾病等。对肾脏和其他器官的损害被认为是由于胱氨酸在各种身体组织的细胞内的积累。这种化学物质也聚集在角膜中，角膜是覆盖在瞳孔和虹膜上的眼睛。10到20年后，一些患者的角膜变得如此拥挤，以至于表面可能变得不规则，偶尔会导致小的，痛苦的破裂。

Trial of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis Clinical Protocol support contract designed to attain extramural support for developing, designing, interpreting, and evaluating clinical trials, epidemiologic and natural history studies. In addition, it will provide for outcomes research involving eye diseases and visual disorders and some preclinical studies. The focus shall be on the design of studies and the collection, analysis, and interpretation of data emanating from these studies, as well as support, and monitoring patient safety and follow-up. Contractor shall also provide analytical and data management support, as described in the work statement, for specified clinical research data bases, cost-effectiveness and economic analyses, quality of life assessment and outcomes research. This will include, but not be limited to, the following areas: analysis of Medicare and other health care databases; evaluation of existing NEI databases such as, centralized NEI Intramural Research database, the Eye Disease Case Control Study, Early Treatment Diabetic Retinopathy Study, Framingham Eye Study, and intramural AIDS and uveitis databases. Objective of Clinical Protocol: This study will continue to evaluate the long term safety and effectiveness of cysteamine eye drops for treating cystine crystals in the corneas of patients with cystinosis. These drops are not sold commercially and are available only through this study. New patients may enroll in the study to obtain them. Cystinosis is an inherited disease that results in poor growth and kidney disease, among other things. The damage to the kidneys and other organs is thought to be due to accumulation of cystine inside the cells of various body tissues. This chemical also accumulates in the cornea-the covering of the eye over the pupil and iris. After 10 to 20 years, the corneas of some patients become so packed with crystals that the surfaces may become irregular, occasionally causing small, painful breaks.