Duchenne Muscular Dystrophy Quality of Life: Qualitative Interviews With Patients and Caregivers
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 68
- 试验地点
- 1
- 主要终点
- Qualitative Interview
研究概览
简要总结
The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with DMD and/or and their caregivers.
This is a qualitative interview study in which individuals with DMD and/or their caregivers will be asked to participate in a semi-structured, approximately 60- minute interview. Interviews will focus on functional abilities and independence. Caregivers and boys with DMD will be interviewed. This study includes no treatment nor intervention; however, some participants are being treated by a drug that is approved in the U.S. and the U.K. and under investigation in other geographies.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subsample A - Patients
- •Eligible participants for Subsample A must:
- •Have been treated with Givinostat for at least 2 years
- •Be at least 10 years of age with signed consent of a parent or legal guardian
- •Be currently taking givinostat
- •Willing and able to participate in a video and/or audio recorded interview
- •Subsample A - Caregivers
- •Eligible caregivers for Subsample A must:
- •Be a parent or legal guardian of an individual with DMD that has been taking givinostat for at least two years
- •Willing and able to participate in a video and/or audio recorded interview
- •Subsample B - Caregivers
- •Eligible caregivers for Subsample B must:
- •Provide care to an individual with DMD who is unable to raise their hands above their head as confirmed by the caregiver
- •Reside in the United States or Canada
- •Able to read, speak, and understand English
- •Willing and able to participate in a video and/or audio recorded interview Have access to a stable internet connection
排除标准
- 未提供
结局指标
主要结局
Qualitative Interview
时间窗: 60 minutes
Participants will participate in a semi-structured interview
次要结局
未报告次要终点