Natural History Study in Prodromal and Manifest Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 70
- 试验地点
- 10
- 主要终点
- DDR gene expression
研究概览
简要总结
SHIELD HD is an international, multisite, prospective, longitudinal cohort natural history study to assess the natural history of HD and its biomarkers that are associated with modulation of the number of cytosine-adenine-guanine (CAG) repeats in the mutant Huntingtin (HTT) gene.
Approximately 60 patients will be enrolled into the study and followed for up to 24 months at clinical sites in North America and Europe.
The results of this study will inform assessments for a future interventional treatment trial.
详细描述
The rationale for this study is to obtain longitudinal information related to Somatic Instability and DNA damage response genes in HDGECs at various stages of the disease. Established assessments of disease progression will also be recorded.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 63 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
DDR gene expression
时间窗: 2 years
To assess deoxyribonucleic acid (DNA) damage repair (DDR) gene expression in accessible biofluids and disease trajectories for established and novel biomarkers and clinical outcomes.
次要结局
- Compare rates of change in biomarkers for disease progression(2 years)