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

NCT03680365

Last updated date
Study Location
Engage Health, Inc.
Eagan, Minnesota, 55121, United States
Contact
1-800-718-1021

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Duchenne Muscular Dystrophy, Dependency Burden, Disability Physical, Disease Management, Impairment, Rare Diseases
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
11 + years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


1. Inability to meet any of the inclusion criteria

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Duchenne Muscular Dystrophy, Dependency Burden, Disability Physical, Disease Management, Impairment, Rare DiseasesYour Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
NCT03680365
  1. Eagan, Minnesota
ALL GENDERS
11 Years+
years
MULTIPLE SITES
Advanced Information
Descriptive Information
Brief Title Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
Official Title Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of DMD on the Lives of Families
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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Cross-Sectional
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition
  • Duchenne Muscular Dystrophy
  • Burden, Dependency
  • Disability Physical
  • Disease Management
  • Impairment
  • Rare Diseases
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: March 29, 2019)
60
Original Estimated Enrollment
 (submitted: September 20, 2018)
120
Actual Study Completion Date March 15, 2019
Actual Primary Completion Date March 15, 2019   (Final data collection date for primary outcome measure)
Eligibility 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

Sex/Gender
Sexes Eligible for Study:All
Ages 11 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers Yes
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03680365
Other Study ID Numbers Jett 0001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD:Undecided
Responsible Party Jett Foundation, Inc.
Study Sponsor Jett Foundation, Inc.
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, Inc.
  • Hoffmann-La Roche
  • Pfizer
  • Capricor Inc.
  • NS Pharma, Inc.
Investigators
Principal Investigator:Christine McSherry, R.N.Jett Foundation, Inc.
PRS Account Jett Foundation, Inc.
Verification Date March 2019