DMD Voice: Qualitative Interviews With Patients and Caregivers
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy (DMD). Basic parameters: from 10 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Duchenne Muscular Dystrophy Quality of Life: Qualitative Interviews With Patients and Caregivers
Overview
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.
Primary outcome measures
- Qualitative Interview [Time frame: 60 minutes]
Eligibility 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
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Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- Red Nucleus — Yardley
Identifiers
NCT: NCT06925269 · CAS-ITF001-01