Extracellular Vesicles As Non-Invasive Biomarkers for Huntington Disease Progression and Huntingtin Lowering Therapy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- To discover blood-based biomarker of brain HTT protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials.
研究概览
简要总结
The primary objective of this study is to discover blood-based biomarker of brain Huntingtin (HTT) protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials.
Secondary objectives of this study include developing more accurate biomarkers of Huntington disease (HD) progression or conversion and to develop standard practices for extracellular vesicle biomarker discovery research.
The investigators hypothesize that brain-derived extracellular vesicles (EVs) isolated from human biofluids contain biological cargo specific to their tissue of origin that could allow their use as brain biomarkers for HD. EVs are lipid bilayer-delimited particles that are naturally released from cells in the brain.
The investigators will investigate if EVs contents reflect the pathological alterations occurring with disease progression when compared with EVs isolated from biofluids of healthy non-HD persons.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •18 - 75 years of age
- •can provide informed consent
- •able to read and speak English
- •agree to comply with study procedures (including overnight fasting, blood collection and lumbar puncture); and
- •has been diagnosed with HD (HD Carriers) or not been diagnosed with HD (Non-HD Carriers).
排除标准
- •younger than 18 or older than 75 years old
- •known to carry an intermediate CAG repeat between 27 and 39 or a larger expansion of 60 or more CAG repeats
- •receiving nutrition through a tube
- •participated in a clinical drug trial within 30 days
- •use prescribed or non-prescribed medications that are not compatible with collection of the study samples (those that may cause excessive bleeding or prevent clotting)
- •positive for HIV, hepatitis B or C
- •have a confirmed or suspected immunodeficient condition/state
- •significant medical, psychiatric, or neurological morbidity is observed by the clinic physician on the day of sample collection that might impair completion of the study procedures
- •have needle phobia, frequent headache, significant lower spinal deformity or major surgery
- •received antiplatelet or anticoagulant therapy within 14 days prior to sample collection (including but not limited to: aspirin, clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban and apixaban)
- •have a blood clotting or bruising disorder
- •do not comply with or are unwilling to undertake any of the study procedures
结局指标
主要结局
To discover blood-based biomarker of brain HTT protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials.
时间窗: 1 year
次要结局
- To develop more accurate biomarkers of HD progression or conversion(8 years)
- to develop standard practices for extracellular vesicle biomarker discovery research(8 years)