DMD Voice: Qualitative Interviews With Patients and Caregivers

April 29, 2025 updated by: Red Nucleus Enterprise Solutions, LLC

Duchenne Muscular Dystrophy Quality of Life: 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.

Study Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

68

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Pennsylvania
      • Yardley, Pennsylvania, United States, 19067
        • Recruiting
        • Red Nucleus
        • Contact:
        • Principal Investigator:
          • Mindy Leffler

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.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Caregivers of individuals with Duchenne Muscular Dystrophy (DMD) and patients with DMD

Description

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:

-

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Subsample A - Caregivers
Caregivers of individuals with DMD treated with givinostat for at least 2 years
Subsample A - Patients
Patients with DMD treated with givinostat for at least 2 years
Subsample B - Caregivers
Caregivers of individuals with DMD never treated with givinostat

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Qualitative Interview
Time Frame: 60 minutes
Participants will participate in a semi-structured interview
60 minutes

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Red Nucleus Enterprise Solutions, LLC

Collaborators

Italfarmaco S.A

Investigators

  • Principal Investigator: Mindy Leffler, M.Ed., Red Nucleus Solutions

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 31, 2025

Primary Completion (Estimated)

December 1, 2025

Study Completion (Estimated)

December 1, 2026

Study Registration Dates

First Submitted

April 3, 2025

First Submitted That Met QC Criteria

April 10, 2025

First Posted (Actual)

April 13, 2025

Study Record Updates

Last Update Posted (Actual)

April 30, 2025

Last Update Submitted That Met QC Criteria

April 29, 2025

Last Verified

April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAS-ITF001-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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