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

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.

Conditions

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

Sponsors & Collaborators

• Engage Health Inc.

  collaborator INDUSTRY

• Hyman, Phelps, & McNamara, P.C.

  collaborator UNKNOWN

• Ryans Quest Inc.

  collaborator UNKNOWN

• Michaels Cause Inc.

  collaborator UNKNOWN

• Nationwide Children's Hospital

  collaborator OTHER

• Solid Biosciences Inc.

  collaborator INDUSTRY

• Santhera Pharmaceuticals

  collaborator INDUSTRY

• Italfarmaco

  collaborator INDUSTRY

• Catabasis Pharmaceuticals

  collaborator INDUSTRY

• Wave Life Sciences Ltd.

  collaborator INDUSTRY

• Sarepta Therapeutics, Inc.

  collaborator INDUSTRY

• Hoffmann-La Roche

  collaborator INDUSTRY

• Pfizer

  collaborator INDUSTRY

• Capricor Inc.

  collaborator INDUSTRY

• NS Pharma, Inc.

  collaborator INDUSTRY

• Jett Foundation, Inc.

  lead OTHER

Principal Investigators

• Christine McSherry, R.N. · Jett Foundation, Inc.

Eligibility

Min Age

11 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2018-09-20

Primary Completion

2019-03-15

Completion

2019-03-15

Countries

• United States

Study Locations