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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

60 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-09-20

Study Completion Date

2019-03-15

Brief Summary

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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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Detailed Description

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Conditions

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

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

CROSS_SECTIONAL

Eligibility Criteria

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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)

Minimum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes