Interviews with patients and caregivers about Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Quality of Life: Qualitative Interviews With Patients and Caregivers
This study is talking to people with Duchenne Muscular Dystrophy and their caregivers to hear about their daily lives and how they feel about their independence and quality of life.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 68 (estimated) |
| Ages | 10 Years and up |
| Sex | Male |
| Sponsor | Red Nucleus Enterprise Solutions, LLC Industry-sponsored |
| Locations | 1 site (Yardley, Pennsylvania) |
| Trial ID | NCT06925269 on ClinicalTrials.gov |
What this trial studies
This observational study aims to gather qualitative insights from individuals with Duchenne Muscular Dystrophy (DMD) and their caregivers regarding functional abilities, independence, and quality of life. Participants will engage in semi-structured interviews lasting approximately 60 minutes, focusing on their experiences and perspectives. The study does not involve any treatment or intervention but includes participants currently receiving a drug called givinostat, which is approved in some regions and under investigation elsewhere.
Who should consider this trial
Good fit: Ideal candidates include boys aged 10 and older who have been treated with givinostat for at least two years.
Not a fit: Patients who are not currently taking givinostat or do not meet the age and treatment duration criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of DMD's impact on daily life, potentially informing future care strategies and interventions.
How similar studies have performed: While qualitative studies on DMD exist, this specific approach focusing on caregiver and patient perspectives in relation to givinostat is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 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 Criteria: \-
Where this trial is running
Yardley, Pennsylvania
- Red Nucleus — Yardley, Pennsylvania, United States (Recruiting)
Study contacts
- Principal investigator: Mindy Leffler, M.Ed. — Red Nucleus Solutions
- Study coordinator: Mary Hubbard
- Email: mhubbard@rednucleus.com
- Phone: 904-541-9326
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.