DMD Voice: Qualitative Interviews With Patients and Caregivers

Red Nucleus Enterprise Solutions

Status

Enrolling

Conditions

Duchenne Muscular Dystrophy (DMD)

Study type

Observational

Funder types

Industry

Identifiers

NCT06925269

CAS-ITF001-01

Details and patient eligibility

About

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.

Enrollment

68 estimated patients

Sex

Male

Ages

10+ years old

Volunteers

No Healthy Volunteers

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