Trial of Cysteamine in Treatment of Corneal Cystine Accumulation in Cystinosis

半胱胺治疗胱氨酸病角膜胱氨酸蓄积的试验

• 批准号: 8075893
• 负责人: DARBY THOMPSON
• 金额: $ 0.66万
• 依托单位: THE EMMES COMPANY, LLC
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-11-01 至 2011-10-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8075893
• 关键词: Acquired Immunodeficiency Syndrome; Area Analyses; Case-Control Studies; Cells; Chemicals; Clinical Research; Clinical Trials; Collection; Contractor; Contracts; Cornea; Cysteamine; Cystine; Cystinosis; Data; Databases; Diabetic Retinopathy; Disease; Drops; Early treatment; Economics; Effectiveness; Enrollment; Epidemiology; Evaluation; Extramural Activities; Eye; Eye diseases; Eyedrops; Growth; Healthcare; Inherited; Intramural Research; Iris; Kidney; Kidney Diseases; Medicare; Monitor; Natural History; Organ; Outcomes Research; Pain; Patients; Pupil; Quality-of-Life Assessment; Research Design; Safety; Specific qualifier value; Surface; Tissues; Uveitis; Vision Disorders; Work; cost effectiveness; data management; design; follow-up; patient safety; preclinical study

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.

局部半胱胺治疗半胱氨酸病角膜半胱氨酸结晶积存的试验