Duchenne Muscular DystrophyNCT03951675
The Burden of Access in Duchenne Muscular Dystrophy in the US
An observational study of Duchenne Muscular Dystrophy, run by University of Florida at 1 site in United States.
Trial summary
This study is being conducted to determine if DMD patients / families and healthcare providers experience burdens related to access, and if so, to identify them, and to determine life impacts to the patient, if any, of these burdens. Read more about this study
- Who is it for?
- Ages 18 to 99 yearsHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 57 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Jun 2019to 28 May 2020
- 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 18 to 99 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: For Patient/Parent Interviews: Patients residing in the US who have been diagnosed with DMD who are age 18 years or older, or the parent / legal guardian of a person of any age who has been diagnosed with DMD For Healthcare Provider Survey: Healthcare providers (physicians, nurse practitioners, and physician assistants) currently involved in the care of patients with DMD
Who may be able to join
- Patient/Parent interviews
- Patients residing in the US who have been diagnosed with DMD who are age 18 years or older, or the parent / legal guardian of a person of any age who has been diagnosed with DMD,
- Have provide "Proof of DMD" to ensure that they are impacted by the disease,
- Who have provided sufficient information in the RSVP process to determine their functional status; ambulatory, transitional or non-ambulatory,
- State that they are the person who deals with insurance issues for the affected patient and,
- Who are able to understand and consent to participation in the study
Show all 12Show fewer
- Healthcare Provider survey
- Healthcare providers (physicians, nurse practitioners, and physician assistants) currently involved in the care of patients with DMD
- Are currently practicing in the US,
- Who have provided sufficient information in the survey screening to determine that they currently care for DMD patients,
- State that they and/or persons on their staff interface with insurance companies for DMD patients related to access to medications, services and/or medical equipment and,
- Who are able to understand and consent to participation in the study
Who cannot join
- There are no stated exclusion criteria in this study. Study population must meet all inclusion criteria in order to be deemed eligible to participate.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Burden of Access in Duchenne Muscular Dystrophy in the US. A Qualitative Assessment of the Impact of Access on the Lives of Families Affected by DMD and Their Healthcare Providers.
This study is being conducted to determine if DMD patients / families and healthcare providers experience burdens related to access, and if so, to identify them, and to determine life impacts to the patient, if any, of these burdens. Data from healthcare providers will be collected by an online survey and from patients/families by one on one telephone interview.
Read the detailed description
Patient/Parent Portion
In this non-interventional study, DMD patients / parents of DMD patients will be interviewed to gather qualitative input, in the patient's voice, regarding challenges associated with access to medications, services and medical equipment, and how these burdens impact quality of life.
Healthcare Provider Portion
In this non-interventional study, healthcare providers who see patients with DMD and who deal with insurance issues on behalf of DMD patients will participate in an online survey designed to determine the burden associated with access to medications, services and medical equipment.
- Conditions
- Duchenne Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Individuals Living with DMD | 90 patients/parents | |
| Healthcare Providers to Patients with DMD | 40 healthcare providers |
What is being measured
The primary outcome is the main question the study is designed to answer.
Burden frequency by functional category
The frequency that each burden is mentioned by a patient according to their functional category as measured by the VIGNOS scale. This is the number of times each burden is mentioned by a patient during their interview, assessed by each functional category.
Measured: Over 12 months
Secondary outcomes (3)
Burden frequency by type of insurance
The frequency that each burden is mentioned by a patient according to their insurance coverage. This is the number of times each burden is mentioned by a patient during their interview, assessed by each type of insurance.
Measured: Over 12 months
Life impact frequency by functional category
The frequency that each life impact is mentioned by a patient according to their functional category as measured by the VIGNOS scale. This is the number of times each life impact is mentioned by a patient during their interview, assessed by each functional category.
Measured: Over 12 months
Life impact frequency by type of insurance
The frequency that each life impact is mentioned by a patient according to their insurance coverage. The number of times each life impact is mentioned by a patient during their interview, assessed by each type of insurance.
Measured: Over 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2019
- Study start18 Jun 2019
- Primary completion28 May 2020
- Study completion28 May 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- 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-control
- Time perspective
- Cross-sectional
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Engage Health Inc.
- Responsible party
- Sponsor
- Investigators
- Kara Godwin, MSN,APRN, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03951675
- Sponsor's protocol ID
- OCR21561
- Other IDs
- LHF0001
- Status last verified
- Aug 2020
- First submitted
- 14 May 2019
- Last updated
- 6 Aug 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Burden of Access in Duchenne Muscular Dystrophy in the US. A Qualitative Assessment of the Impact of Access on the Lives of Families Affected by DMD and Their Healthcare Providers.
- ClinicalTrials.gov ID
- NCT03951675
- Acronym / sponsor ID
- OCR21561
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Engage Health Inc.
- Study start
- 18 Jun 2019 (actual)
- Primary completion
- 28 May 2020 (actual)
- Study completion
- 28 May 2020 (actual)
- First posted
- 15 May 2019 (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.
This study is being conducted to determine if DMD patients / families and healthcare providers experience burdens related to access, and if so, to identify them, and to determine life impacts to the patient, if any, of these burdens. Data from healthcare providers will be collected by an online survey and from patients/families by one on one telephone interview.
Detailed description
Patient/Parent Portion
In this non-interventional study, DMD patients / parents of DMD patients will be interviewed to gather qualitative input, in the patient's voice, regarding challenges associated with access to medications, services and medical equipment, and how these burdens impact quality of life.
Healthcare Provider Portion
In this non-interventional study, healthcare providers who see patients with DMD and who deal with insurance issues on behalf of DMD patients will participate in an online survey designed to determine the burden associated with access to medications, services and medical equipment.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Individuals Living with DMD | – | –90 patients/parents |
| Healthcare Providers to Patients with DMD | – | –40 healthcare providers |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- For Patient/Parent Interviews: Patients residing in the US who have been diagnosed with DMD who are age 18 years or older, or the parent / legal guardian of a person of any age who has been diagnosed with DMD For Healthcare Provider Survey: Healthcare providers (physicians, nurse practitioners, and physician assistants) currently involved in the care of patients with DMD
Key inclusion criteria (12 listed)
- Patient/Parent interviews
- Patients residing in the US who have been diagnosed with DMD who are age 18 years or older, or the parent / legal guardian of a person of any age who has been diagnosed with DMD,
- Have provide "Proof of DMD" to ensure that they are impacted by the disease,
- Who have provided sufficient information in the RSVP process to determine their functional status; ambulatory, transitional or non-ambulatory,
- State that they are the person who deals with insurance issues for the affected patient and,
Key exclusion criteria (1 listed)
- There are no stated exclusion criteria in this study. Study population must meet all inclusion criteria in order to be deemed eligible to participate.
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
Burden frequency by functional category
Time frame: Over 12 months
The frequency that each burden is mentioned by a patient according to their functional category as measured by the VIGNOS scale. This is the number of times each burden is mentioned by a patient during their interview, assessed by each functional category.
3 secondary outcomes
Burden frequency by type of insurance
Time frame: Over 12 months
Life impact frequency by functional category
Time frame: Over 12 months
Life impact frequency by type of insurance
Time frame: Over 12 months
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 6 Aug 2020.
- Individual participant dataSharing planned: No
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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.
Kara Godwin, MSN,APRN principal investigator
University of Florida
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, run by University of Florida 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 12 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 6 Aug 2020. 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?
Burden frequency by functional category (time frame: Over 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 Aug 2020.