GLP Analogs for Diabetes in Wolfram Syndrome Patients
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 主要终点
- beta cell function
研究概览
简要总结
Wolfram syndrome, also referred to as DIDMOAD (diabetes insipidus, diabetes mellitus, optic atrophy and deafness) is a genetic syndrome characterized by beta-cell dysfunction and apoptosis leading to diabetes, neurodegeneration and psychiatric illness. Accumulating evidence indicates that beta-cell failure and neuronal cell dysfunction in Wolfram's syndrome results from a high level of ER stress in affected cells. The current treatment of Wolfram syndrome is insulin, which fails to prevent the progression of beta-cell failure.
Several studies showed that GLP-1 analogs are very effective in protecting beta-cells from ER stress. Herein, the investigators suggest studying the impact of GLP-1 analogs in the treatment of patients with Wolfram syndrome.
The investigators will Study the effects of GLP-1 analog (Exanatide) on beta-cell function and glycemic control of patients with Wolfram syndrome. Evaluation of beta cell function will be done by performing meal test and IVGTT test before starting GLP-1 therapy, and after 3 month of treatment.
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
- •Genetic or definitive clinical diagnosis of Wolfram's syndrome including: diabetes mellitus, optic atrophy and at least one additional neurological dysfunction (diabetes insipidus, sensorineural deafness, neurogenic bladder or other type of autonomic or peripheral neuropathy)
- •Age >18 years
- •Duration of diabetes of <10 years.
排除标准
- •pregnant women
- •patients who are unable to give inform consent.
研究组 & 干预措施
Exenatide
干预措施: Exenatide (Drug)
结局指标
主要结局
beta cell function
时间窗: 3 months
IVGTT test and meal test will be performed before starting treatment with Exenetide and after 3 months of treatment.
次要结局
未报告次要终点
研究者
Adar Zinger
Physician
Hadassah Medical Organization