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

study id #: NCT03680365

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

status: recruiting

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.

mechanism of action: No pharmaceutical intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT03680365

last updated: January 08, 2019

start date: September 20, 2018

estimated completion: June 2019

size / enrollment: 120

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

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

sponsor: Jett Foundation, Inc.

contacts: Bridget Knisely, (651) 994-0510, bknisely@engagehealth.com; Patti A. Engel (B.S., R.N.), (651) 994-0510, pengel@engagehealth.com

investigators: Christine McSherry R.N.

locations: United States