PROSPECT: A Registry and Longitudinal Natural History Study of Individuals at Risk for Genetic Prion Disease
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 2,000
- 试验地点
- 1
- 主要终点
- Disease Onset or Death
研究概览
简要总结
PROSPECT is a prospective observational registry and longitudinal natural history study of adults at risk for genetic prion disease.
详细描述
The study collects genetic, cognitive, neuropsychiatric, symptom, and survival data to characterize disease onset and progression in individuals carrying or at risk for PRNP mutations. Information from this registry may support future biomarker development, prevention trials, and therapeutic studies in genetic prion disease
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Age ≥18 years
- •At risk for genetic prion disease based on family history
- •Willing to comply with all study procedures including genetic testing and longitudinal follow-up
- •Resident in the United States
- •Sufficiently proficient in English to participate in all study procedures
排除标准
- •Lacking capacity to independently consent at time of initial enrollment
- •Symptomatic of prion disease at time of initial enrollment
- •Inability to provide a backup contact
研究组 & 干预措施
Individuals at risk for Genetic Prion Disease
Adults age 18 years and older who are at risk for genetic prion disease based on family history and enrolled in the PROSPECT longitudinal natural history registry.
结局指标
主要结局
Disease Onset or Death
时间窗: Baseline and annual follow-up assessments for up to 10 years after enrollment.
To determine the occurrence of disease onset or death among individuals at risk for genetic prion disease according to PRNP genotype.
次要结局
未报告次要终点
研究者
Brian Appleby
Physician
University Hospitals Cleveland Medical Center