DMD Voice: Qualitative Interviews With Patients and Caregivers

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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Study Overview

Study Details

Study 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.

Duchenne Muscular Dystrophy Quality of Life: Qualitative Interviews With Patients and Caregivers

DMD Voice: Qualitative Interviews With Patients and Caregivers

Condition
Duchenne Muscular Dystrophy (DMD)
Intervention / Treatment

-

Contacts and Locations

Yardley

Red Nucleus, Yardley, Pennsylvania, United States, 19067

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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
  • * 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
  • * 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

Ages Eligible for Study

10 Years to

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Red Nucleus Enterprise Solutions, LLC,

Mindy Leffler, M.Ed., PRINCIPAL_INVESTIGATOR, Red Nucleus Solutions

Study Record Dates

2026-12