A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 100
- 试验地点
- 4
- 主要终点
- volumetric magnetic resonance imaging
研究概览
简要总结
In the study the investigators plan to include 100 early affected HD patients (5 ≤ UHDRS ≤ 40) in a randomized, double-blind, controlled study in 2 centers (France and the Netherlands). Patients will receive either triheptanoin at 1g/kg of body weight per day (n = 50), or a control oil (n = 50) at 1g/kg of body weight per day for 6 months followed by an open label phase using triheptanoin for another 6 months. Efficacy of triheptanoin will be evaluated by measurements of caudate volume using volumetric magnetic resonance imaging. Clinical improvement will be evaluated by brain energy metabolism quantification as evaluated by the ratio of inorganic phosphate/phosphocreatine, during visual stimulation, using 31P-MRS, UHDRS, TFC, and PBA-S scores as well as performance on the neuropsychological battery. Patient quality of life will be evaluated with the SF-36 questionnaire before and after treatment; biological tolerance and compliance will be evaluated by routine biochemical blood tests, plasma and urine measurements of triheptanoin oil derivatives and patient report.
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
- •Positive genetic test with CAG repeat length ≥39 in HTT gene
- •At least 18 years of age
- •Signature of informed consent
- •Covered by social security
- •UHDRS score between 5 and 40
- •Ability to undergo MRI scanning
- •BMI between 18 and 30
排除标准
- •Hypersensitivity to triheptanoin or to one of its excipients
- •Additional major comorbidities
- •History of severe head injury
- •Participation in another therapeutic trial (3 month exclusion period)
- •For women of childbearing age, the absence of two forms of effective contraception (with the exception of those who are abstinent)
- •Pregnancy or breastfeeding
- •Inability to understand information about the protocol
- •Persons deprived of their liberty by judicial or administrative decision
- •Adult subject under legal protection or unable to consent
- •Treatment with tetrabenazine
研究组 & 干预措施
Placebo group
Subjects will receive safflower oil at 1g/kg/day for 6 months (months 0 - 6) and triheptanoin oil at 1g/kg/day for 6 months (months 7 - 12)
干预措施: Placebo (Drug)
Triheptanoin group
Subjects will receive triheptanoin oil at 1g/kg/day for 6 months (months 0 - 6) and triheptanoin oil at 1g/kg/day for 6 months (months 7 - 12)
干预措施: Triheptanoin oil (Drug)
结局指标
主要结局
volumetric magnetic resonance imaging
时间窗: 6 months
A decrease in the rate of caudate atrophy, using volumetric MRI
次要结局
- 31- Phosphorus Magnetic Resonance Spectroscopy(3 months)
- Patient autonomy after 6 months(6 months)
- Sustained restoration of brain energy metabolism after 12 months(12 months)
- Caudal Atrophy(12 months)
- Patient autonomy after 12 months(12 months)
- Sustained restoration of brain energy metabolism after 6 months(6 months)
- Trail making test after 12 months(12 months)
- Digit span test after 6 months(6 months)
- motor function after 6 months(6 months)
- motor function after 12 months(12 months)
- Symbol Digit Modalities Test after 6 months(6 months)
- Digit span test after 12 months(12 months)
- patients' daily life(6 months)
- quality of life questionnaire after 6 months(6 months)
- quality of life questionnaire after 12 months(12 months)
- Symbol Digit Modalities Test after 12 months(12 months)
- Stroop test after 6 months(6 months)
- Trail making test after 6 months(6 months)
- Stroop test after 12 months(12 months)
- psychiatric symptoms after 3 months(3 months)
- psychiatric symptoms after 6 months(6 months)
- psychiatric symptoms after 9 months(9 months)
- psychiatric symptoms after 12 months(12 months)
- Number of adverse events(12 months)
- clinical exam for Long term tolerance(12 months)
- home nurse visits for Long term tolerance(12 months)