NCT06925269 · Red Nucleus Enterprise Solutions, LLC
DMD Voice: Qualitative Interviews With Patients and Caregivers
What this study is about
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.
View original scientific 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. 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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
Where
- Yardley, Pennsylvania
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 30, 2025 · Source of record for eligibility and locations