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NCT Number: NCT06925269

DMD Voice: 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.

Recruiting

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Key information

Conditions

Duchenne Muscular Dystrophy (DMD) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

10 year and older

Sex eligibility

Male

Study type

Observational

Primary location

Red Nucleus

Yardley, Pennsylvania, 19067, United States

Location status: Recruiting

Location contact

Mary Hubbard

CONTACT

[email protected]

904-541-9326

Mindy Leffler

PRINCIPAL_INVESTIGATOR

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

-

Treatment and study plan

Primary outcomes

  1. Qualitative Interview

    Time frame: 60 minutes

    Participants will participate in a semi-structured interview

Study contacts

Contact information is provided by the study sponsor or research team.

Mary Hubbard

CONTACT

[email protected]

904-541-9326

Sponsors and collaborators

Lead sponsor

Red Nucleus Enterprise Solutions, LLC

Industry

Collaborators

  • Italfarmaco S.A

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2025
Study completion
2026
First posted
Apr 13, 2025
Registry last updated
Apr 30, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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