Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 553
- 试验地点
- 47
- 主要终点
- Change in Total Functional Capacity
研究概览
简要总结
Huntington's disease (HD) is a slowly progressive disorder that devastates the lives of those affected and their families. There are no treatments that slow the progression of HD, only mildly effective symptomatic therapies are available.Creatine monohydrate is considered a nutritional supplement. The purpose of CREST-E is to test whether high-dose creatine can slow the progressive functional decline that occurs in persons 18 years or older with early clinical features of HD. The long-term safety, tolerability and effectiveness of up to 40 grams daily creatine compared to placebo is studied. A variety of biological processes are assessed for markers of disease activity or progression and creatine effects. Up to 50 active research centers globally will enroll 650 subjects.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Male or female ages 18 or older.
- •Clinical features of HD AND confirmatory family history of HD; OR Clinical features of HD AND CAG repeat expansion greater or equal to
- •Stage I or II of illness (TFC greater or equal to 7).
- •Ambulatory and not requiring skilled nursing care at the time of enrollment.
- •Must be capable of providing informed consent and complying with trial procedures.
- •Additional inclusion criteria apply.
排除标准
- •History of known sensitivity or intolerability to creatine monohydrate.
- •Exposure to any investigational drug within 30 days of randomization (Baseline visit).
- •Use of supplemental creatine at a dose greater than 10 grams within 30 days of randomization (Baseline visit).
- •Screening laboratory abnormalities that in the judgment of the investigator would jeopardize safe conduct of study.
- •Clinical evidence of unstable medical illness.
- •Clinical evidence of unstable psychiatric illness.
- •Additional exclusion criteria apply.
研究组 & 干预措施
A
Randomized to receive creatine monohydrate (up to 40 grams daily)
干预措施: Creatine Monohydrate (Drug)
B
Randomized to receive placebo (up to 40 grams daily)
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Total Functional Capacity
时间窗: Minimum 12 months up to 48 months
Study duration depends on each subject's calendar date of enrollment.
次要结局
- Clinical symptoms (changes in other UHDRS scores); safety (frequency of adverse events); tolerability (proportion of subjects completing study at assigned dosage level), quality of life, other biological markers.(Duration of the trial)
研究者
Steven M. Hersch
Professor of Neurology
Massachusetts General Hospital