PWS Outcome Assessment Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 59
- 试验地点
- 1
- 主要终点
- Meaningful Change in Symptom Categories
研究概览
简要总结
This is a longitudinal study during which qualitative interviews will be conducted with caregivers of Soleno C601/C602 study subjects. There is an additional option for caregivers to collect video data of PWS patients doing specific activities of daily life.
The purpose of this study is to understand the real-world and nuanced impact of a potential therapeutic on individual PWS patients. The results of this study will complement the outcomes being captured during the Soleno C601/C602 clinical studies.
There is no treatment or intervention associated with this study.
研究设计
- 研究类型
- Observational
- 观察模型
- Family Based
- 时间视角
- Other
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
- •Key Inclusion Criteria for Interviews:
- •Eligible caregivers must:
- •Be willing and able to provide informed consent in English
- •Care for a C601/C602 trial subject or potential trial subject
- •Have access to a smartphone or the internet
- •Be able to converse in English
- •Key Inclusion Criteria for Video Capture:
- •Eligible PWS participants for the optional daily life video capture must:
- •Be willing and able to provide informed consent or assent.
- •Be enrolled in the C601/C602 trial
- •Be able to converse in English
- •Have a caregiver who has access to an Apple or Android smartphone or has internet access and is willing to use a wi-fi-only device to record and upload videos
- •Have a caregiver who is willing and able to record activity videos
排除标准
- 未提供
结局指标
主要结局
Meaningful Change in Symptom Categories
时间窗: From baseline for interview #2 (Week 13-16), From baseline for interview #3 (Week 26-28), From baseline for interview #4 (Week 65-67)
The percentage of caregivers reporting meaningful change in each of the symptom categories in the Study C601 treated arm versus the Study C601 placebo arm as measured by the five-point quantification scale anchored in the qualitative questions (Scale: 1=very meaningful negative change, 2=slightly meaningful negative change, 3=no meaningful change, 4=slightly meaningful change, 5=very meaningful positive change)
次要结局
未报告次要终点