Clinical trial NCT03680365: Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
Official title: Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of DMD on the Lives of Families
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Duchenne Muscular Dystrophy
Burden, Dependency
Disability Physical
Disease Management
Impairment
Rare Diseases
Keywords: Duchenne, DMD
Lead sponsor: Jett Foundation, Inc.
Sponsor class: OTHER
Collaborators: name: Engage Health Inc.
name: Hyman, Phelps, & McNamara, P.C.
name: Ryans Quest Inc.
name: Michaels Cause Inc.
name: Nationwide Children's Hospital
name: Solid Biosciences Inc.
name: Santhera Pharmaceuticals
name: Italfarmaco
name: Catabasis Pharmaceuticals
name: Wave Life Sciences Ltd.
name: Sarepta Therapeutics, Inc.
name: Hoffmann-La Roche
name: Pfizer
name: Capricor Inc.
name: NS Pharma, Inc.
First posted: 2018-09-21
Enrollment: 60
Sex: ALL
Minimum age (years): 11
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_ONLY
Time perspective: CROSS_SECTIONAL
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.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Patient/Parent Interview Assessing Treatment Needs
timeFrame: 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Eagan
country: United States
facility: Engage Health, Inc.
state: Minnesota
Study officials:
affiliation: Jett Foundation, Inc.
name: Christine McSherry, R.N.
role: PRINCIPAL_INVESTIGATOR
References:
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.
citation: 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.
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.