Longitudinal Measurement of Synaptic Density to Monitor Progression of Huntington's Disease.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 33
- 试验地点
- 2
- 主要终点
- Correlations between progression of the clinical scores and decline of synaptic density.
研究概览
简要总结
AIM: To assess synaptic density and to investigate the potential relationship of regional synaptic loss with motor and non-motor symptoms and with disease progression in the human brain in vivo in patients with HD.
DESIGN: The investigators will include 20 HD mutations carriers and 15 healthy controls. All subjects will undergo a clinical examination, with comprehensive assessment of motor and non-motor symptoms, and imaging evaluation consisting of 11C-UCB-J PET-CT and 18F-FDG PET-MR at baseline and after 2 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 20 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Age 20-75 years.
- •Capacity to understand the informed consent form.
- •For HD group: CAG repeat expansion in HTT ≥
- •Premanifest HD mutation carriers:
- •* No clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score <
- •Early manifest HD patients:
- •Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score =
- •UHDRS-TFC score 7 or higher (Shoulson-Fahn stage 1 and 2).
排除标准
- •neuropsychiatric diseases other than HD
- •major internal medical diseases
- •white matter lesion load on FLAIR Fazekas score 2 or higher or other relevant MRI abnormalities
- •history of alcohol abuse or current alcohol abuse (chronic use of more than 15 units per week) or drug abuse
- •contraindications for MR
- •pregnancy
- •previous participation in other research studies involving ionizing radiation with >1 mSv in the previous 12 months.
结局指标
主要结局
Correlations between progression of the clinical scores and decline of synaptic density.
时间窗: Data analysis wel be done when all subjects have undergone the 2-year follow-up evaluation.
Correlations between progression of the clinical scores and decline of synaptic density in the patient group, after longitudinal follow up of 2 years.
Baseline correlations between clinical scores and regional synaptic density.
时间窗: Data analysis wel be done when all subjects have undergone the baseline evaluation.
Correlations between clinical scores and regional synaptic density in the patient group at baseline.
Baseline differences in synaptic density.
时间窗: Data analysis wel be done when all subjects have undergone the baseline evaluation.
Baseline differences (%) in (regional) synaptic density between patients and controls.
Differences in the rate of decline of synaptic density.
时间窗: Data analysis wel be done when all subjects have undergone the 2-year follow-up evaluation.
Differences (%) in the rate of decline of synaptic density between patients and controls.
次要结局
- Differences in the rate of decline of cerebral glucose metabolism.(Data analysis wel be done when all subjects have undergone the 2-year follow-up evaluation.)
- Baseline correlations between clinical scores and cerebral glucose metabolism.(Data analysis wel be done when all subjects have undergone the 2-year follow-up evaluation.)
- Baseline differences in cerebral glucose metabolism.(Data analysis wel be done when all subjects have undergone the baseline evaluation.)
- Correlations between progression of the clinical scores and decline of cerebral glucose metabolism in the patient group, after longitudinal follow up of 2 years.(Data analysis wel be done when all subjects have undergone the 2-year follow-up evaluation.)