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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03680365
• Other study ID #: Jett 0001
• Status: Completed
• Start date: September 20, 2018
• Est. completion date: March 15, 2019

Study information

• Verified date: March 2019
• Source: Jett Foundation, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: March 15, 2019
• Est. primary completion date: March 15, 2019
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 11 Years and older

Eligibility

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

Related Conditions & MeSH terms

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

Locations

Country	Name	City	State
United States	Engage Health, Inc.	Eagan	Minnesota

Sponsors (16)

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

Country where clinical trial is conducted

United States, 

References & Publications (3)

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. Review. — View Citation

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. Review. Erratum in: Lancet Neurol. 2018 Apr 4;:. — View Citation

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. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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: Number of times each activity is mentioned; Overall score for each activity; Number of times each reason is mentioned; Overall score for each reason	1 year

External Links

•   Jett Foundation