The Burden of Access in Duchenne Muscular Dystrophy in th... | NCT03951675

Studies
The Burden of Access in Duchenne Muscular Dystrophy in the US

NCT03951675

Sponsor

University of Florida

Status

Completed

Last Update Posted

Aug 6, 2020Actual

Enrollment

57Actual

Phase

Not provided

Conditions

Duchenne Muscular Dystrophy

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
LHF0001	Other Identifier	WIRB

Status Module

Record Verification Date

Aug 2020

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 18, 2019Actual

Primary Completion Date

May 28, 2020Actual

Completion Date

May 28, 2020Actual

First Submitted Date

May 14, 2019

First Submission Date that Met QC Criteria

May 14, 2019

First Posted Date

May 15, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 4, 2020

Last Update Posted Date

Aug 6, 2020Actual

Name	Class
Engage Health Inc.	INDUSTRY

Description Module

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

57Actual

Label	Type	Description	Intervention Names
Individuals Living with DMD		90 patients/parents
Healthcare Providers to Patients with DMD		40 healthcare providers

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Measure	Description	Time Frame
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.	Over 12 months
Life impact frequency by functional category

Other Outcomes

Not provided

Eligibility Module

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.

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

99 Years

Standard Ages

AdultOlder 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

Sampling Method

Probability Sample

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases