Patient and Caregiver Views on Duchenne Muscular Dystrophy Daily Abilities
Interviews with boys with Duchenne muscular dystrophy and their caregivers about daily abilities and independence.
Plain-language title generated from the registry record
Registry title: DMD Voice: Qualitative Interviews With Patients and Caregivers
Key facts
- Where
- Yardley, United StatesSee the site
Where in United States
- Red NucleusYardley, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Red Nucleus Enterprise Solutions, LLC
- Trial code
- NCT06925269
- Record updated
- 30 April 2025
- Original description
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.Read full original descriptionShow less
Duchenne Muscular Dystrophy Quality of Life: Qualitative Interviews With Patients and Caregivers
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.
Inclusion: 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.
Exclusion: -.