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Completed

NCT Number: NCT03951675

The Burden of Access in Duchenne Muscular Dystrophy in the US

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.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

18 year–99 year

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Florida

Gainesville, Florida, 32610, United States

About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

University of Florida

Other

Collaborators

  • Engage Health Inc.

Registry information

Official study 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.

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
May 15, 2019
Registry last updated
Aug 6, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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