Duchenne Muscular DystrophyNCT03680365
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
An observational study of Duchenne Muscular Dystrophy and Burden, Dependency, run by Jett Foundation, Inc. at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 11 years and olderHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Sep 2018to 15 Mar 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 11 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: The study will be conducted in individuals 11 years or older living with Duchenne Muscular Dystrophy as well caregivers.
Who may be able to join
- 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)
Who cannot join
- 1. Inability to meet any of the inclusion criteria
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
- Conditions
- Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management, Impairment, Rare Diseases
- Keywords
- Duchenne, DMD
- Medical subject headings
- Muscular Dystrophy, Duchenne, Rare Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2018
- Study start20 Sep 2018
- Primary completion15 Mar 2019
- Study completion15 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jett Foundation, Inc.
- Sponsor type
- University, hospital or other
- Collaborators
- Engage Health Inc., Hyman, Phelps, & McNamara, P.C., Ryans Quest Inc., Michaels Cause Inc., Nationwide Children's Hospital, Solid Biosciences Inc., Santhera Pharmaceuticals, Italfarmaco, Catabasis Pharmaceuticals, Wave Life Sciences USA, Inc., Sarepta Therapeutics, Inc., Hoffmann-La Roche, Pfizer, Capricor Inc., NS Pharma, Inc.
- Responsible party
- Sponsor
- Investigators
- Christine McSherry, R.N., Principal investigator, Jett Foundation, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03680365
- Sponsor's protocol ID
- Jett 0001
- Status last verified
- Mar 2019
- First submitted
- 6 Sep 2018
- Last updated
- 1 Apr 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 29395989Birnkrant 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.
PubMed 29395990Birnkrant 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.
PubMed 29398641
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of DMD on the Lives of Families
- ClinicalTrials.gov ID
- NCT03680365
- Acronym / sponsor ID
- Jett 0001
- Conditions
- Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management, Impairment, Rare Diseases
- Keywords
- Duchenne, DMD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Jett Foundation, Inc.University, hospital or other
- Collaborators
- Engage Health Inc., Hyman, Phelps, & McNamara, P.C., Ryans Quest Inc., Michaels Cause Inc., Nationwide Children's Hospital, Solid Biosciences Inc., Santhera Pharmaceuticals, Italfarmaco, Catabasis Pharmaceuticals, Wave Life Sciences USA, Inc., Sarepta Therapeutics, Inc., Hoffmann-La Roche, Pfizer, Capricor Inc., NS Pharma, Inc.
- Study start
- 20 Sep 2018 (actual)
- Primary completion
- 15 Mar 2019 (actual)
- Study completion
- 15 Mar 2019 (actual)
- First posted
- 21 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 11 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study will be conducted in individuals 11 years or older living with Duchenne Muscular Dystrophy as well caregivers.
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (1 listed)
- 1. Inability to meet any of the inclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2019.
- Background: PubMed 29395989Birnkrant 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.
- Background: PubMed 29395990Birnkrant 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.
- Background: PubMed 29398641Birnkrant 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.
- Jett FoundationLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christine McSherry, R.N. principal investigator
Jett Foundation, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy and Burden, Dependency, run by Jett Foundation, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Patient/Parent Interview Assessing Treatment Needs (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03680365, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Apr 2019.