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The Burden of Access in Duchenne Muscular Dystrophy in the US

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ClinicalTrials.gov Identifier: NCT03951675
Recruitment Status : Recruiting
First Posted : May 15, 2019
Last Update Posted : June 20, 2019
Sponsor:
Collaborator:
Engage Health, Inc.
Information provided by (Responsible Party):
University of Florida

Brief 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. Data from healthcare providers will be collected by an online survey and from patients/families by one on one telephone interview.

Condition or disease
Duchenne Muscular Dystrophy

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.


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Study Type : Observational
Estimated Enrollment : 130 participants
Observational Model: Case-Control
Time Perspective: Cross-Sectional
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.
Actual Study Start Date : June 18, 2019
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2020


Group/Cohort
Individuals Living with DMD
90 patients/parents
Healthcare Providers to Patients with DMD
40 healthcare providers



Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Burden frequency by type of insurance [ Time Frame: Over 12 months ]
    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.

  2. Life impact frequency by functional category [ Time Frame: Over 12 months ]
    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.

  3. Life impact frequency by type of insurance [ Time Frame: Over 12 months ]
    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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 99 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Probability Sample
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

Criteria

Inclusion Criteria:

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

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

Exclusion Criteria:

  • There are no stated exclusion criteria in this study. Study population must meet all inclusion criteria in order to be deemed eligible to participate.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03951675


Contacts
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Contact: Stephanie M Salabarria, BHSc 3522736582 ssalabarria@ufl.edu

Locations
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United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Stephanie M Salabarria, BHSc       ssalabarria@ufl.edu   
Contact: Kara E Godwin, ARNP       godwinke@ufl.edu   
Sponsors and Collaborators
University of Florida
Engage Health, Inc.
Investigators
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Principal Investigator: Kara Godwin, MSN,APRN University of Florida

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Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT03951675     History of Changes
Other Study ID Numbers: OCR21561
LHF0001 ( Other Identifier: WIRB )
First Posted: May 15, 2019    Key Record Dates
Last Update Posted: June 20, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked