NBIAready: Online Collection of Natural History Patient-reported Outcome Measures
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 300
- 试验地点
- 1
- 主要终点
- Document the natural history of NBIA and identify new markers of disease progression.
研究概览
简要总结
The purpose of this study is to learn more about Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders. Data is being collected on three types of NBIA disorders:
Pantothenate Kinase-Associated Neurodegeneration (PKAN), PLA2G6-associated Neurodegeneration (PLAN) and Beta-propeller Protein-associated Neurodegeneration (BPAN). The study will (1) collect information about how symptoms and findings in NBIA change over time and (2) identify measures of NBIA that can be used in future clinical trials. Participants will follow links to a secure website every 6 months for a period of 5-10 years to electronically complete a set of rating scales as related to their NBIA disorder.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of PKAN, PLAN or BPAN confirmed by gene testing and/or clinical features.
- •Ability to access a computer with internet services or a phone approximately once every 6 months for up to 10 years to enter data.
排除标准
- •Individuals who are not fluent in reading and communicating in English.
结局指标
主要结局
Document the natural history of NBIA and identify new markers of disease progression.
时间窗: 5-10 years
The unit of analyses will be the data collected on NBIA milestones and patient-reported outcome measures. Latent Growth Curve (LGC) modeling will be used to characterize the natural history of NBIA. If data support that certain disease milestones occur in a consistent order, or at consistent ages, then these data points will be candidate markers of disease progression. LGC will be used to characterize the natural history of NBIA and identify areas in which individuals with NBIA differ from the general population.
次要结局
未报告次要终点
研究者
Susan J. Hayflick
Professor & Chair, Molecular & Medical Genetics
Oregon Health and Science University