CYSTEAMINE VS. CYSTAMINE TREATMENT OF CORNEAL CRYSTAL ACCUMULATION

半胱胺对比

• 批准号: 2574536
• 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/2574536
• 关键词: adolescence (12-20); aminothiol; child (0-11); clinical research; conjunctivitis; cornea disorder; cysteamine; cystinosis; drug adverse effect; eye agent; eye disorder chemotherapy; eye pharmacology; eyelid disorder; human subject; human therapy evaluation; laboratory rabbit; nonhuman therapy evaluation; sulfides

A successful, controlled clinical trial of cysteamine vs. placebo has proved the efficacy of cysteamine eyedrops in the treatment of removing crystals from patients with nephropathic cystinosis. Because cysteamine preparation is dispensed at room temperature and 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; clinical efficacy can be determined only through limited human clinical trials. Preceding this, however, experience with toxicity was necessary, and a study was performed in rabbits to determine the range of toxicity of the disulfide compound prior to its use in a clinical trial. 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 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对照临床试验