Preparing for Prevention of Huntington's Disease (PREVENT-HD)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 258
- 试验地点
- 1
- 主要终点
- Unified Huntington's Disease Rating Scale (UHDRS) Total Functional Capacity
研究概览
简要总结
This is a prospective investigation which aims to address key challenges to the design of clinical trials to prevent the onset of Huntington's disease (HD). The project will provide necessary psychometric data for clinical outcome assessments (COAs) and biomarkers (BMs) in the cerebral spinal fluid (CSF) to address questions of central importance to the success of these measures for premanifest clinical trials. Of the 258 participants: 52 will be low risk of motor diagnosis, 102 high risk of motor diagnosis, 52 with diagnosed HD (stages I or II), and 52 healthy controls. Participants can expect to be in the study for up to 2 years.
详细描述
This is a prospective investigation which aims to address key challenges to the design of clinical trials to prevent the onset of Huntington's disease (HD). The project will provide necessary psychometric data for clinical outcome assessments (COAs) and biomarkers (BMs) in the cerebral spinal fluid (CSF) to address questions of central importance to the success of this measure for premanifest clinical trials such as:
- How reliable the measure is in the same person when repeated over time;
- how reliable the measure is in the same person when analyzed by two different labs/sites;
- how well the measure reflects disease symptoms;
- how well the measure predicts meaningful disease outcomes;
- how well the measure tracks disease progression or severity; AND
- how many research subjects are required to test that an intervention is delaying/slowing the onset of HD?
Answers to these questions will better position the field to more effectively test new interventions to prevent HD such as gene therapies and new drugs.
Neurocognitive, motor and behavioral data, blood, CSF, and genetic samples, and MRI measures will be collected from 258 participants at baseline. Of the 258 participants: 52 will be low risk of motor diagnosis, 102 high risk of motor diagnosis, 52 with diagnosed HD (stages I or II), and 52 healthy controls. Neurocognitive and behavioral data, blood and CSF samples, and MRI will be repeated 2 years (18-24 months) after the baseline visit. Remote assessments of clinical outcome measures (neurocognitive, motor and behavioral data) will be conducted as needed.
Specific Study Aims and Methods
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Unified Huntington's Disease Rating Scale (UHDRS) Total Functional Capacity
时间窗: 2 years
UHDRS Total Functional Capacity is a clinician-rating scale of independence in activities of daily living. 13 is fully functioning and any drop in points in noted during pre-diagnosed HD.
Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Level
时间窗: 2 years
UHDRS Motor Diagnosis of HD Diagnostic Confidence Level is a clinical rating of how confident the movement disorder specialist is that the person has manifest HD with over 99% confidence; scale is 0-4 where 0 is 'normal' and 4 is the highest motor dysfunction.
Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score
时间窗: 2 years
UHDRS Total Motor Score is a 31-item instrument each item scored on a scale of 0-4 where 0 is 'normal' and 4 is the highest motor dysfunction. Total possible range of scores is 0-124.
次要结局
- CANTAB composite score(baseline, 1 year, 2 years)
- Cognitive Assessment Battery (CAB) Composite Score(baseline, 1 year, 2 years)
- Tablet Cognitive Assessment Total (TabCat) Score(baseline, 1 year, 2 years)
- Problem Behavior Assessment - short form (PBA) Score(baseline, 1 year, 2 years)