DMD Voice: Qualitative Interviews With Patients and Caregivers

NCT06925269 | Recruiting

• 1 other identifier: CAS-ITF001-01
• Study Type: observational
• Target: 68
• Locations: 1 country
• Sites: 1

Brief Summary

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)

Keywords

Duchenne; givinostat

Outcome Measures

Primary Outcomes (1)

• Qualitative Interview

  Participants will participate in a semi-structured interview

  60 minutes

Study Arms (3)

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

Eligibility Criteria

• Age: 10 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

Sponsors & Collaborators

• Red Nucleus Enterprise Solutions, LLC: lead
• Italfarmaco S.A: collaborator

Study Sites (1)

Red Nucleus

Yardley, Pennsylvania, 19067, United States

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Mindy Leffler, M.Ed.

  Red Nucleus Solutions

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mary Hubbard

CONTACT

904-541-9326; mhubbard@rednucleus.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 3, 2025
• First Posted: April 13, 2025
• Study Start: March 31, 2025
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: April 30, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)