CLINICAL TRIAL OF NEW DRUG THERAPY FOR CYSTINOSIS

半胱胺研究提供了药物问题的答案 疗效，但有一定的推论难度，因为半胱胺令人不快 味道和气味使许多患者不喜欢它，他们随后 没有收到有效剂量的药物。这项研究的设计 本身，没有随机同时对照组。、遮蔽效果和 在决赛中需要很大程度上依赖于调整技术 分析。 本研究的目的是改进对胱氨酸病的治疗，并 确定半胱胺的更多影响。在药物开发阶段 对半胱胺类似物，磷酸半胱胺的研究， 据透露，它会在血液中迅速转化为半胱胺，因此 这两种药是有效的等效药。此外，由于口感和 对于一些患者来说，磷酸半胱胺的气味不那么令人讨厌，它起到了 作为一种替代治疗，可能会改善患者的依从性。这个 目前的研究将患者随机分成低剂量的半胱胺(或 磷酸半胱胺，由患者选择)或高剂量；如此设计 试验是确定最佳治疗方案的最佳工具 治疗。 在签约学习中心协调患者招募和治疗 在加州大学圣地亚哥分校。数据中心的功能是 位于达拉斯的德克萨斯大学健康科学中心的责任。 这项研究将包括至少3-4年的登记和治疗 90个病人。治疗将在肾脏的基础上进行评估 功能通过血清肌酐水平和内生肌酐清除量来衡量， 作为肾小球滤过率的替代物，在研究结束时。

The Cysteamine Study provided answers to the question of the drug's efficacy with some inferential difficulty, since cysteamine's unpleasant taste and smell rendered it unpalatable to many patients, who subsequently did not receive effective amounts of the drug. The design of the study itself, with no randomized concurrent control group., obscured effects and required a good deal of reliance on adjustment techniques in the final analysis. The object of the current study is to improve treatment of cystinosis and determine more of the effects of cysteamine. In the drug development phase of the trial, investigation of a cysteamine analog, phosphocysteamine, revealed that it converts rapidly to cysteamine in the bloodstream, so that the two drugs are effective equivalents. Moreover, since the taste and smell of phosphocysteamine are less obnoxious to some patients, it serves as an alternative treatment that may improve patient compliance. The current study randomizes patients to a low dose of cysteamine (or phosphocysteamine as the patient chooses) or to a high dose; so designed the trial is an optimal vehicle for ascertaining the best course of treatment. Patient recruitment and treatment is coordinated at contracted study center at the University of California, San Diego. Data center functions are the responsibility of the University of Texas Health Science Center at Dallas. The study will encompass 3-4 years of enrollment and treatment of at least 90 patients. The treatments will be evaluated on the basis of renal function as measured by serum creatinine levels and creatinine clearance, as a surrogate of glomerular filtration rate, at the end of the study.