CYSTEAMINE VS. CYSTAMINE TREATMENT OF CORNEAL CRYSTAL ACCUMULATION

半胱胺对比

• 批准号: 5203267
• 负责人: M KAISER-KUPFER
• 金额: --
• 依托单位: NATIONAL EYE INSTITUTE
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/5203267
• 关键词: aminothiol; child (0-11); clinical trials; conjunctivitis; cornea disorder; crystallization; cysteamine; cystinosis; disulfide bond; dosage; drug adverse effect; eye agent; eye disorder chemotherapy; eye pharmacology; eyelid disorder; histopathology; human subject; human therapy evaluation; laboratory rabbit; nonhuman therapy evaluation; sulfides; temperature

A successful controlled clinical trial of cysteamine vs. placebo has proven the efficacy of cysteamine eyedrops in removing crystals from patients with nephropathic cystinosis. Because th cysteamine preparation is dispensed at room temperature and because room temperature cysteamine oxidizes the disulfide cysteamine, it has been suggested that this more stable compound be studied for safety and efficacy. To date, there have been no studies on the clinical efficacy of the disulfide, although current speculation is that the compound will be pharmacologically active at similar concentrations. No animal model for cystinosis exists; therefore, clinical efficacy can be determined only through limited human clinical trials. Before trials, however, experience with toxicity was necessary, and a study was performed in rabbits to determine the range of toxicity of the disulfide compound. Twenty-four rabbits were observed in a double-blind study for ocular toxicity testing of cysteamine disulfide, cystamine. Complete data are available, but, in summary, the reaction to 0.5 percent and 1 percent was essentially no different from the placebo. Cysteamine 2.0 percent showed both clinical and histopathologic evidence of blepharitis and conjunctivitis. This study is to perform a randomized, controlled, double-masked clinical trial of cysteamine vs. cystamine for equivalency in removing crystals from the cornea and in maintaining a "crystal- free" or near crystal-free cornea.

一项成功的半胱胺与 安慰剂已经证明了半胱胺滴眼液在 从肾病患者体内取出晶体 胱氨酸病 因为半胱胺制剂被分配给 因为室温下的半胱胺 氧化二硫化物半胱胺， 研究这种更稳定的化合物的安全性， 功效 到目前为止，还没有关于 二硫化物的临床疗效，虽然目前 据推测，该化合物将被 在相似的浓度下活跃。 无动物模型 存在胱氨酸病;因此，临床疗效可以 仅通过有限的人体临床试验确定。 然而，在试验之前， 必要的，并在兔子中进行了研究， 确定二硫化物化合物的毒性范围。 24只家兔进行了双盲研究 对于半胱胺二硫化物的眼毒性测试， 胱胺 完整的数据是可用的，但是，总的来说， 0.5%和1%的反应基本上是 与安慰剂没有区别。 半胱胺2.0% 临床和组织病理学证据显示 睑炎和结膜炎。 这项研究是为了执行 一项随机、对照、双盲临床试验， 半胱胺与胱胺在去除 从角膜和维持一个"晶体- 游离或接近无晶体角膜。