Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)

Status unconfirmed

Conditions studied: Duchenne Muscular Dystrophy

In brief

The purpose of this study is to establish the largest long-term assessment of people with Duchenne muscular dystrophy (DMD). In this study, the investigators associated with the Cooperative International Neuromuscular Research Group CINRG) will take a detailed look (for a minimum of eight years) at DMD participant's physical abilities, the medical problems they experience, and how they use health care services. Physical abilities will be compared to a group of healthy controls. The second purpose of this study is to find out whether small, normal differences in the genetic makeup of people with DMD (called "single nucleotide polymorphisms" or "SNPs") affect how their disease progresses and relates to muscle strength/size and steroid response. The third purpose of this study is to study genetic variations associated with DMD. The final purpose of this study is to determine whether certain biomarkers are present in people with DMD and not in healthy controls.

Key facts

Study ID

NCT00468832

Run by

Cooperative International Neuromuscular Research Group

People needed

551

Starts

2005-12-01

Expected to finish

2019-12-01

Last updated by the study team

2016-04-21

Who can join

Age: 2 and older, up to 30. Sex: male. Healthy volunteers: accepted.

Where it is running

• University of California, Davis — Sacramento, California, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• University of of Florida — Gainesville, Florida, United States
• Lurie Children's Hospial — Chicago, Illinois, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington University, St. Louis — St Louis, Missouri, United States
• Carolinas Medical Center — Charlotte, North Carolina, United States
• Duke Children's Hospital — Durham, North Carolina, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• University of Tennessee — Memphis, Tennessee, United States
• Children's Hospital of Virginia — Richmond, Virginia, United States
• Fundacion Favaloro — Buenos Aires, Argentina
• The Children's Hospital at Westmead — Sydney, New South Wales, Australia
• Royal Children's Hospital — Melbourne, Victoria, Australia
• Alberta Children's Hospital — Calgary, Alberta, Canada
• University of Alberta — Edmonton, Alberta, Canada
• Holland Bloorview Kids Rehab Hospital — Toronto, Ontario, Canada
• Apollo Hospitals — Chennai, India
• Schneider Children's Medical Center of Israel — Petach Tikvah, Israel
• NEMO — Milan, Italy
• Queen Silvia Children's Hospital — Gothenburg, Sweden

Full record on ClinicalTrials.gov

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