Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

March 29, 2019 updated by: Jett Foundation, Inc.

Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of DMD on the Lives of Families

The purpose of this study is to improve the understanding of the treatment goals that a person with Duchenne Muscular Dystrophy (DMD) or the caregiver may be most interested in, based on the severity of the person's disease. Data will be collected by online survey when the participant accepts the study invitation ("RSVP questionnaire") and telephone interview on the functional burden and self-identified treatment goals from the perspective of people with DMD and their caregivers. Interviews will be analyzed to help identify things important to Duchenne families to measure in clinical trials and to inform the selection of key concepts of interest and development of future clinical outcome measures, including observer reported outcomes/patient reported outcomes. The study will be conducted in the United States and will enroll between 45 and 120 participants 11 years or older living with DMD as well as their caregivers. The time commitment for the online survey and the telephone interview is about one hour. It is anticipated that the entire study will be completed within one year.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

60

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Minnesota
      • Eagan, Minnesota, United States, 55121
        • Engage Health, Inc.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

11 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The study will be conducted in individuals 11 years or older living with Duchenne Muscular Dystrophy as well caregivers.

Description

Inclusion Criteria:

  1. Participant must be a person with DMD who is 11 years or older or The parent/legal guardian of a person with DMD who is under the age of 18 years.
  2. Confirmed diagnosis of DMD with written proof of disease provided
  3. Resident of the U.S.
  4. Able to read, write and communicate in English
  5. Able to grant informed consent
  6. Willing to participate in a 45 minute telephone interview
  7. Ability to view or receive a document from the interviewer before or during the interview (web browser, ability to receive a text, fax or document by mail)

Exclusion Criteria:

1. Inability to meet any of the inclusion criteria

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Patient/Parent Interview Assessing Treatment Needs
Time Frame: 1 year

In this non-interventional study, up to 120 patients/parents will participate in an online survey designed to determine the patient's functional category; ambulatory, transitional, or non-ambulatory. 15 patients from each functional category will be interviewed to gather qualitative input, in the patient's voice, regarding activities they would like to do but cannot do because of DMD, and reasons why these activities are important to them. Qualitative responses will be scored to provide quantitative frequency counts and point values for each answer dependent upon if the response was the most important, 2nd most important and 3rd most important activity to the participant. Data will be coded by two independent coders to ensure consistency. Scores will be calculated by functional category for:

  1. Number of times each activity is mentioned
  2. Overall score for each activity
  3. Number of times each reason is mentioned
  4. Overall score for each reason
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Jett Foundation, Inc.

Collaborators

Pfizer
Hoffmann-La Roche
Italfarmaco
Nationwide Children's Hospital
Capricor Inc.
Santhera Pharmaceuticals
Sarepta Therapeutics, Inc.
NS Pharma, Inc.
Catabasis Pharmaceuticals
Wave Life Sciences Ltd.
Engage Health Inc.
Solid Biosciences Inc.
Hyman, Phelps, & McNamara, P.C.
Ryans Quest Inc.
Michaels Cause Inc.

Investigators

  • Principal Investigator: Christine McSherry, R.N., Jett Foundation, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

September 20, 2018

Primary Completion (ACTUAL)

March 15, 2019

Study Completion (ACTUAL)

March 15, 2019

Study Registration Dates

First Submitted

September 6, 2018

First Submitted That Met QC Criteria

September 20, 2018

First Posted (ACTUAL)

September 21, 2018

Study Record Updates

Last Update Posted (ACTUAL)

April 1, 2019

Last Update Submitted That Met QC Criteria

March 29, 2019

Last Verified

March 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Jett 0001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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