Ursodiol in Huntington's Disease
试验速览
- 阶段
- 1 期
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Safety measures (complete blood count, chemistry profile, electrocardiogram, urinalysis)
研究概览
简要总结
The purpose of this study is to evaluate the safety of the drug ursodiol (ursodeoxycholic acid, UDCA) in people with Huntington's disease (HD) and to explore how the compound is processed by the body.
详细描述
Huntington's disease is an inherited neurodegenerative disease that causes a movement disorder, dementia, and psychiatric and behavioral disturbance in affected individuals.
Tauroursodeoxycholic acid (TUDCA) is a bile acid synthesized in the liver by the conjugation of taurine to ursodeoxycholic acid (UDCA). It is thought to function as an anti-apoptotic agent in HD, evidenced by studies in toxic cell models and both toxic and transgenic rodent models of the disease.
Ursodiol is a commercially-available exogenous form of UDCA, the precursor of TUDCA. Although the compound has an established dosing, safety, tolerability and efficacy profile in patients with hepatobiliary disorders, gaps exist in the understanding of the pharmacokinetics / pharmacodynamics of the compound, particularly in patients with normal gastrointestinal function, and no human data exist for its therapeutic use in neurodegenerative disorders. The specific aims of this study are:
- To establish whether treatment with the drug ursodiol will result in measurable levels of its bile acid metabolites in serum and CSF at standard oral doses; and whether a dose-response can be detected using these measures.
- To establish a preliminary safety and tolerability profile of the drug in subjects with HD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
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
- •All subjects will be age 18 or older
- •All subjects will have manifest Huntington disease determined by clinical exam plus either documented prior DNA testing for the HD gene or a documented family history of the disease
排除标准
- •Subjects taking oral contraceptives, cholestyramine, colestipol, or aluminum-based antacids will be excluded
- •Subjects with known allergy or other contraindication to the study drug will be excluded
- •Subjects with bleeding diathesis, or on coumadin or mandatory aspirin will be excluded
- •Subjects with unstable medical or psychiatric illness will be excluded
- •Subjects with clinically significant lab / EKG abnormalities at screening will be excluded
- •Subjects who are currently pregnant or breastfeeding will be excluded
研究组 & 干预措施
A
干预措施: ursodiol (Drug)
B
干预措施: ursodiol (Drug)
C
干预措施: placebo (Drug)
结局指标
主要结局
Safety measures (complete blood count, chemistry profile, electrocardiogram, urinalysis)
时间窗: 35 days
Tolerability measures (adverse event severity)
时间窗: 35 days
Pharmacokinetic measures (Serum and CSF levels of bile acids)
时间窗: 28 days
次要结局
未报告次要终点