A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 50
- 主要终点
- measurement of liver enzymes in blood tests
研究概览
简要总结
The assumption is that in some of the carriers, the increase in enzymes reflects tissue damage due to excess copper. The reduction of the amount of copper absorbed will decrease excess copper in the liver, which will result in a decrease in the level of liver enzymes. Zinc causes the induction of metalothionines in the intestine, which in turn prevents absorption of copper from the digestive system. Zinc administration in Wilson's patients causes the depletion of copper deposits and constitutes one of the cornerstones in the treatment of this disease.
详细描述
The research group is composed of patients over the age of 18 referred for unexplained elevation of liver enzymes and carry a single mutation in the ATP7B gene. After a washout period of 3 months these patients will be re-checked for liver enzymes and if high will receive zinc therapy at a dose of 300 mg / day for 6 months, after which the liver enzymes will be checked again.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patients over the age of 18 unexplained elevation of liver enzymes patients that carry a single mutation in the ATP7B gene.
排除标准
- 未提供
研究组 & 干预措施
unexplained elevation of liver enzymes
patients over the age of 18 referred for unexplained elevation of liver enzymes and carry a single mutation in the ATP7B gene. After a washout period of 3 months these patients will be re-checked for liver enzymes and if high will receive zinc therapy at a dose of 300 mg / day for 6 months, after which the liver enzymes will be checked again.
干预措施: Zinc (Dietary Supplement)
结局指标
主要结局
measurement of liver enzymes in blood tests
时间窗: 9 months
A model based on the assumption that at least 50 subjects will be recruited, and assuming that 50% of the patients will have a significant reduction of liver enzymes, is statistically significant and supports the association between a single mutation in the ATP7B gene and liver injury.
次要结局
未报告次要终点
研究者
Prof. Elon Pras
head of The Institute of Human Genetics
Sheba Medical Center