Purpose:

The primary goal of this study is to establish the largest long-term assessment of people with Duchenne muscular dystrophy (DMD) between the ages of 2 and 30 years. Study participants will be enrolled for five years. CINRG sites will collect details on patient’s physical abilities across all ages, medical problems they experience, and how they use healthcare services over this five year period. A second goal is to look for an association between genes (which contain instructions for how the body works) and response to treatment of DMD.

Further Study Details:

There are 22 sites (U.S. and International) participating in this observational study through the Cooperative International Neuromuscular Research Group (CINRG). This is a five year study collecting information from boys and men with DMD and their families. This study will collect information including:

• Strength and lung function tests.
• Meet with a study doctor for an exam.
• Have an interview and complete study questionnaires.
• Blood OR saliva samples will be collected ONCE at the first study visit.

During study visits family members of boys/men with DMD will:

• Complete study questionnaires

Study visits will include 10 visits total:

Year 1: Four visits total (visits every three months)
Year 2: Two visits total (visits every six months)
Years 3, 4 & 5: One visit each year.

Participants have the choice to take part in other research projects during their enrollment in this observational study.

TARGET NUMBER OF PARTICIPANTS: 395

RECRUITMENT STATUS: Open

Subject Inclusion Criteria:

Participants with DMD must:

• Male
• Between the ages of 2 and 30
• Have a confirmed diagnosis of DMD (This will be confirmed as a part of the screening and enrollment process)

Subject Exclusion Criteria:

• Participants who are currently enrolled in a clinical trial may not enroll in this study until 2 weeks after they have completed the clinical trial. Once in the study, clinical trial participation IS allowed.

Contact Angela Zimmerman or Erik Henricson for more information on this study or for information on where study sites are located.

CONTACT INFORMATION:

Angela Zimmerman
CINRG Project Manager
111 Michigan Avenue, NW
Washington, DC 20010
Phone: 202-884-4110
Fax: 202-884-6014
Email: azimmerman@cnmcresearch.org
Web: www.cinrgresearch.org


Erik Henricson, MPH
Research Associate
Phone: 916-734-2923
Email: erik.henricson@ucdmc.ucdavis.edu