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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

Tracking Information
First Submitted Date May 14, 2019
First Posted Date May 15, 2019
Last Update Posted Date June 20, 2019
Actual Study Start Date June 18, 2019
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 14, 2019)
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.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03951675 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: May 14, 2019)
  • 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.
  • 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.
  • 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.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title The Burden of Access in Duchenne Muscular Dystrophy in the US
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.
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.
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 Type Observational
Study Design Observational Model: Case-Control
Time Perspective: Cross-Sectional
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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

Condition Duchenne Muscular Dystrophy
Intervention Not Provided
Study Groups/Cohorts
  • Individuals Living with DMD
    90 patients/parents
  • Healthcare Providers to Patients with DMD
    40 healthcare providers
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: May 14, 2019)
130
Original Estimated Enrollment Same as current
Estimated Study Completion Date June 2020
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 99 Years   (Adult, Older Adult)
Accepts Healthy Volunteers Yes
Contacts
Contact: Stephanie M Salabarria, BHSc 3522736582 ssalabarria@ufl.edu
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03951675
Other Study ID Numbers OCR21561
LHF0001 ( Other Identifier: WIRB )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party University of Florida
Study Sponsor University of Florida
Collaborators Engage Health, Inc.
Investigators
Principal Investigator: Kara Godwin, MSN,APRN University of Florida
PRS Account University of Florida
Verification Date June 2019