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

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ClinicalTrials.gov Identifier: NCT03680365
Recruitment Status : Recruiting
First Posted : September 21, 2018
Last Update Posted : September 21, 2018
Sponsor:
Collaborators:
Engage Health, Inc.
Hyman, Phelps, & McNamara, P.C.
Ryans Quest Inc.
Michaels Cause Inc.
Nationwide Children's Hospital
Solid Biosciences, LLC
Santhera Pharmaceuticals
Italfarmaco
Catabasis Pharmaceuticals
Wave Life Sciences Ltd.
Sarepta Therapeutics
Hoffmann-La Roche
Pfizer
Capricor Inc.
NS Pharma, Inc.
Information provided by (Responsible Party):
Jett Foundation, Inc.

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
Duchenne Muscular Dystrophy Burden, Dependency Disability Physical Disease Management Impairment Rare Diseases

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Study Type : Observational
Estimated Enrollment : 120 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 : September 20, 2018
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : June 2019





Primary Outcome Measures :
  1. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   11 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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.
Criteria

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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03680365


Contacts
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Contact: Bridget Knisely (651) 994-0510 bknisely@engagehealth.com
Contact: Patti A. Engel, B.S., R.N. (651) 994-0510 pengel@engagehealth.com

Locations
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United States, Minnesota
Engage Health, Inc. Recruiting
Eagan, Minnesota, United States, 55121
Contact: Bridget Knisely    651-994-0510    bknisely@engagehealth.com   
Contact: Patti A Engel, B.S., R.N.    651-994-0510    pengel@engagehealth.com   
Sponsors and Collaborators
Jett Foundation, Inc.
Engage Health, Inc.
Hyman, Phelps, & McNamara, P.C.
Ryans Quest Inc.
Michaels Cause Inc.
Nationwide Children's Hospital
Solid Biosciences, LLC
Santhera Pharmaceuticals
Italfarmaco
Catabasis Pharmaceuticals
Wave Life Sciences Ltd.
Sarepta Therapeutics
Hoffmann-La Roche
Pfizer
Capricor Inc.
NS Pharma, Inc.
Investigators
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Principal Investigator: Christine McSherry, R.N. Jett Foundation, Inc.

Additional Information:
Publications:
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Responsible Party: Jett Foundation, Inc.
ClinicalTrials.gov Identifier: NCT03680365     History of Changes
Other Study ID Numbers: Jett 0001
First Posted: September 21, 2018    Key Record Dates
Last Update Posted: September 21, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Jett Foundation, Inc.:
Duchenne, DMD

Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Rare Diseases
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Disease Attributes
Pathologic Processes