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

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.

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Key information

Conditions

Duchenne Muscular Dystrophy Burden, Dependency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disability Physical Disease Attributes Disease Management Genetic Diseases, Inborn Genetic Diseases, X-Linked Impairment Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases

Age range

11 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Engage Health, Inc.

Eagan, Minnesota, 55121, United States

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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.
Confirmed diagnosis of DMD with written proof of disease provided
Resident of the U.S.
Able to read, write and communicate in English
Able to grant informed consent
Willing to participate in a 45 minute telephone interview
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

Inability to meet any of the inclusion criteria

Treatment and study plan

Primary outcomes

Patient/Parent Interview Assessing Treatment Needs

Time frame: 1 year
In this non-interventional study, up to 120 patients/parents will participate in an online survey designed to determine the patient's functional category; ambulatory, transitional, or non-ambulatory. 15 patients from each functional category will be interviewed to gather qualitative input, in the patient's voice, regarding activities they would like to do but cannot do because of DMD, and reasons why these activities are important to them. Qualitative responses will be scored to provide quantitative frequency counts and point values for each answer dependent upon if the response was the most important, 2nd most important and 3rd most important activity to the participant. Data will be coded by two independent coders to ensure consistency. Scores will be calculated by functional category for:
- Number of times each activity is mentioned
- Overall score for each activity
- Number of times each reason is mentioned
- Overall score for each reason

Sponsors and collaborators

Lead sponsor

Jett Foundation, Inc.

Other

Collaborators

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

Registry information

Official study title

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

Important dates

Study start: 2018
Primary completion: 2019
Study completion: 2019
First posted: Sep 21, 2018
Registry last updated: Apr 1, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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