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

Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of DMD on the Lives of Families


Lead Sponsor: Jett Foundation, Inc.

Collaborator: Engage Health, Inc.
Hyman, Phelps, & McNamara, P.C.
Ryans Quest Inc.
Michaels Cause Inc.
Nationwide Children's Hospital
Solid Biosciences, LLC
Santhera Pharmaceuticals
Catabasis Pharmaceuticals
Wave Life Sciences Ltd.
Sarepta Therapeutics, Inc.
Hoffmann-La Roche
Capricor Inc.
NS Pharma, Inc.

Source Jett Foundation, Inc.
Brief Summary

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.

Overall Status Completed
Start Date September 20, 2018
Completion Date March 15, 2019
Primary Completion Date March 15, 2019
Study Type Observational
Primary Outcome
Measure Time Frame
Patient/Parent Interview Assessing Treatment Needs 1 year
Enrollment 60

Sampling Method: Non-Probability Sample


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

Gender: All

Minimum Age: 11 Years

Maximum Age: N/A

Healthy Volunteers: Accepts Healthy Volunteers

Overall Official
Last Name Role Affiliation
Christine McSherry, R.N. Principal Investigator Jett Foundation, Inc.
Facility: Engage Health, Inc.
Location Countries

United States

Verification Date

March 2019

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Patient Data Undecided
Study Design Info

Observational Model: Case-Only

Time Perspective: Cross-Sectional