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

NCT03680365 | Completed

• 1 other identifier: Jett 0001
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Keywords

Duchenne, DMD

Outcome Measures

Primary Outcomes (1)

• Patient/Parent Interview Assessing Treatment Needs

  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: 1. Number of times each activity is mentioned 2. Overall score for each activity 3. Number of times each reason is mentioned 4. Overall score for each reason

  1 year

Eligibility Criteria

• Age: 11 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study will be conducted in individuals 11 years or older living with Duchenne Muscular Dystrophy as well caregivers.

You may qualify if:

• 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)

Sponsors & Collaborators

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

Study Sites (1)

Engage Health, Inc.

Eagan, Minnesota, 55121, United States

Location

Related Publications (3)

• Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, Case LE, Clemens PR, Hadjiyannakis S, Pandya S, Street N, Tomezsko J, Wagner KR, Ward LM, Weber DR; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018 Mar;17(3):251-267. doi: 10.1016/S1474-4422(18)30024-3. Epub 2018 Feb 3.

  PMID: 29395989 BACKGROUND

• Birnkrant DJ, Bushby K, Bann CM, Alman BA, Apkon SD, Blackwell A, Case LE, Cripe L, Hadjiyannakis S, Olson AK, Sheehan DW, Bolen J, Weber DR, Ward LM; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. Epub 2018 Feb 3.

  PMID: 29395990 BACKGROUND

• Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Colvin MK, Cripe L, Herron AR, Kennedy A, Kinnett K, Naprawa J, Noritz G, Poysky J, Street N, Trout CJ, Weber DR, Ward LM; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Lancet Neurol. 2018 May;17(5):445-455. doi: 10.1016/S1474-4422(18)30026-7. Epub 2018 Feb 2.

  PMID: 29398641 BACKGROUND

Related Links

• Jett Foundation

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Rare Diseases

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Christine McSherry, R.N.

  Jett Foundation, Inc.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 6, 2018
• First Posted: September 21, 2018
• Study Start: September 20, 2018
• Primary Completion: March 15, 2019
• Study Completion: March 15, 2019
• Last Updated: April 1, 2019

Record last verified: 2019-03

Locations

US (1)