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

Sponsor

Jett Foundation, Inc. (Other)

Overall Status

Completed

CT.gov ID

NCT03680365

Collaborator

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

Enrollment

Location

5.8

Actual Duration (Months)

10.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy Burden, Dependency Disability Physical Disease Management Impairment Rare Diseases

Study Design

Study Type:

Observational

Actual Enrollment :

60 participants

Observational Model:

Case-Only

Time Perspective:

Cross-Sectional

Official Title:

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

Actual Study Start Date :

Sep 20, 2018

Actual Primary Completion Date :

Mar 15, 2019

Actual Study Completion Date :

Mar 15, 2019

Outcome Measures

Primary Outcome Measures

Patient/Parent Interview Assessing Treatment Needs [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: Number of times each activity is mentioned Overall score for each activity Number of times each reason is mentioned Overall score for each reason

Eligibility Criteria

Criteria

Ages Eligible for Study:

11 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

Inclusion Criteria:

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.
Confirmed diagnosis of DMD with written proof of disease provided
Resident of the U.S.
Able to read, write and communicate in English
Able to grant informed consent
Willing to participate in a 45 minute telephone interview
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:

Inability to meet any of the inclusion criteria

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Engage Health, Inc.	Eagan	Minnesota	United States	55121

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Jett Foundation

Publications

Responsible Party:

Jett Foundation, Inc.

ClinicalTrials.gov Identifier:

NCT03680365

Other Study ID Numbers:

Jett 0001

First Posted:

Sep 21, 2018

Last Update Posted:

Apr 1, 2019

Last Verified:

Mar 1, 2019

Individual Participant Data (IPD) Sharing Statement:

Undecided

Plan to Share IPD:

Undecided

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Jett Foundation, Inc.

Duchenne, DMD

Additional relevant MeSH terms:

Muscular Dystrophies

Muscular Dystrophy, Duchenne

Rare Diseases

Study Results

No Results Posted as of Apr 1, 2019