Non-Interventional, Observational, Natural History Study of Serious Medical Events in Prader-Willi Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 700
- 试验地点
- 1
- 主要终点
- Evaluate the incidence of serious medical events
研究概览
简要总结
PATH for PWS is a study to help researchers better understand serious medical events in PWS over a 4-year period, as well as evaluate how PWS-related behaviors change over time. The data from this study is intended to inform the development and clinical trial design of potential new treatments.
详细描述
Every 6 months, participants or their caregivers will be asked to update online surveys about medical problems and serious medical events, as well as provide information about conditions and behaviors often associated with PWS such as hyperphagia. Because this is an observational study, no study drug will be provided and no visits to a doctor or clinic are required.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must meet all of the following criteria:
- •Confirmed diagnosis of PWS
- •At least 5 years of age
- •Live in the United States, Canada, Australia, or New Zealand
- •Must be enrolled or willing to enroll in the Global PWS Registry (https://pwsregistry.org)
- •Participants or their caregiver must also meet all of the following criteria:
- •Have access to the internet to complete online surveys at least every 6 months
- •Agree to receive reminders to complete online surveys
- •Allow the staff to ask questions about survey responses if more information is needed and to enter data into the surveys
排除标准
- •Participants or their caregiver meeting any of the following criteria will be excluded:
- •Is not able to read and understand English
结局指标
主要结局
Evaluate the incidence of serious medical events
时间窗: Up to 4 years
Serious medical events are those that result in death, are life-threatening, require hospitalization or an emergency room visit, or are medically significant
次要结局
- Evaluate food-related behaviors using an online survey(Up to 4 years)
- Analyze D-dimer concentrations in a subset of participants who agree to provide a blood sample(Up to 4 years)
- Evaluate prescription medication use associated with serious medical events and thrombotic events(Up to 4 years)
- Evaluate hyperphagia management using an online survey(Up to 4 years)
- Analyze medical information to evaluate the natural history of PWS(Up to 4 years)
- Evaluate the change in weight(Up to 4 years)
- Evaluate the change in height(Up to 4 years)
- Evaluate the change in height-adjusted weight(Up to 4 years)
- Evaluate PWS complexity using an online survey(Up to 4 years)
- Evaluate the pattern of hyperphagia behaviors using an online survey(Up to 4 years)
- Evaluate the incidence of non-serious thrombotic events(Up to 4 years)