Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
| Status: | Completed |
|---|---|
| Conditions: | Neurology, Orthopedic |
| Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 11 - Any |
| Updated: | 4/3/2019 |
| Start Date: | September 20, 2018 |
| End Date: | March 15, 2019 |
Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of 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.
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.
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
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