NEW DRUG THERAPY IN TREATMENT OF NEPHROPATHIC CYSTINOSIS

治疗肾病性胱氨酸病的新药物疗法

• 批准号: 2314968
• 负责人: JERRY A SCHNEIDER
• 金额: $ 33.33万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 1992
• 资助国家: 美国
• 起止时间: 1992-09-01 至 1995-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/2314968
• 关键词: NEW; DRUG; THERAPY; TREATMENT; NEPHROPATHIC

Cystinosis is a genetic disease which characterized by the renal tubular Fanconi syndrome in infancy, followed by progressive glomerular damage requiring dialysis and transplantation by 10 years of age. Among several other clinical manifestations is severe growth retardation. Cystinosis is treated with a cystine depleting thiol, cysteamine, which in addition to aiding other organs, also increases growth. However, most children with cystinosis still remain well below the 3rd percentile for height. Children with cystinosis are not growth hormone deficient, however, preliminary data in older children show that daily injections of growth hormone does stimulate growth. Though growth improves, there is also evidence that glomerular function may decrease in these older children as they experience increased growth. The design of this clinical trial is to determine if growth hormone will significantly increase growth velocity without having an adverse effect on glomerular function in cystinosis patients. Approximately 30 patients will receive growth hormone and 15 patients will serve as non-placebo controls for 24 months.

囊肿性是一种以肾小管为特征的遗传疾病 Fanconi综合征在婴儿期，然后进行性肾小球损伤 需要透析和移植10岁。 在几个 其他临床表现是严重的生长迟缓。 囊肿病是 用胱氨酸耗尽硫醇，氰氨酸治疗，除了 协助其他器官也会增加增长。 但是，大多数孩子 囊肿性仍然远低于第三百分点的高度。 孩子们 伴有膀胱变性不是生长激素缺乏的，但是，初步数据 在大龄儿童中，每天注射生长激素确实 刺激生长。 尽管增长有所改善，但也有证据表明 这些年龄较大的孩子经历的肾小球功能可能会降低 增长增加。 该临床试验的设计是确定是否 生长激素将显着提高生长速度 对囊肿病患者的肾小球功能的不良影响。 大约30名患者将接受生长激素，15例患者将 用作24个月的非地位对照。