NCT03951675

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
57

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Jun 2019

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 14, 2019

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 15, 2019

Completed
1 month until next milestone

Study Start

First participant enrolled

June 18, 2019

Completed
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 28, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 28, 2020

Completed
Last Updated

August 6, 2020

Status Verified

August 1, 2020

Enrollment Period

12 months

First QC Date

May 14, 2019

Last Update Submit

August 4, 2020

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

  • 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.

    Over 12 months

Secondary Outcomes (3)

  • Burden frequency by type of insurance

    Over 12 months

  • Life impact frequency by functional category

    Over 12 months

  • Life impact frequency by type of insurance

    Over 12 months

Study Arms (2)

Individuals Living with DMD

90 patients/parents

Healthcare Providers to Patients with DMD

40 healthcare providers

Eligibility Criteria

Age18 Years - 99 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodProbability 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

You may qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Florida

Gainesville, Florida, 32610, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Kara Godwin, MSN,APRN

    University of Florida

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 14, 2019

First Posted

May 15, 2019

Study Start

June 18, 2019

Primary Completion

May 28, 2020

Study Completion

May 28, 2020

Last Updated

August 6, 2020

Record last verified: 2020-08

Data Sharing

IPD Sharing
Will not share

Locations

US(1)