Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

Jett Foundation, Inc.

Status: Completed

Conditions: Burden, Dependency; Disease Management; Impairment; Rare Diseases; Duchenne Muscular Dystrophy; Disability Physical

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT03680365

Jett 0001

Details and patient eligibility

About

The purpose of this study is to improve the understanding of the treatment goals that a person with Duchenne Muscular Dystrophy (DMD) or the caregiver may be most interested in, based on the severity of the person's disease. Data will be collected by online survey when the participant accepts the study invitation ("RSVP questionnaire") and telephone interview on the functional burden and self-identified treatment goals from the perspective of people with DMD and their caregivers. Interviews will be analyzed to help identify things important to Duchenne families to measure in clinical trials and to inform the selection of key concepts of interest and development of future clinical outcome measures, including observer reported outcomes/patient reported outcomes. The study will be conducted in the United States and will enroll between 45 and 120 participants 11 years or older living with DMD as well as their caregivers. The time commitment for the online survey and the telephone interview is about one hour. It is anticipated that the entire study will be completed within one year.

Enrollment: 60 patients

Sex: All

Ages: 11+ years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

1. Participant must be a person with DMD who is 11 years or older or The parent/legal guardian of a person with DMD who is under the age of 18 years.
2. Confirmed diagnosis of DMD with written proof of disease provided
3. Resident of the U.S.
4. Able to read, write and communicate in English
5. Able to grant informed consent
6. Willing to participate in a 45 minute telephone interview
7. Ability to view or receive a document from the interviewer before or during the interview (web browser, ability to receive a text, fax or document by mail)

Exclusion criteria

1. Inability to meet any of the inclusion criteria