This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy

Many therapeutic trials in DMD exclude non-ambulatory boys and men. Rate of progression in these non-ambulatory patients has been studied but consensus has not been reached for what measures are most reliable and reproducible. Furthermore, any treatment trial would be expected to demonstrate improved function and improvement in quality of life. Therefore, function, strength, and quality of life must be understood and standardized. While the goal of this proposal is to standardize clinical outcomes for therapeutic trials, careful understanding of the progression of DMD in non ambulatory boys may also lead to better medical treatment.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

7 year–22 year

Sex eligibility

Male

Study type

Observational

Primary location

Washington University

St Louis, Missouri, 63110, United States

About this study

For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.

Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.

Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.

Exclusion criteria

For Non-ambulatory study, the inability to understand and cooperate with the testing would exclude a subject. -

Treatment and study plan

Primary outcomes

Dystrophin gene mutations that predispose to early onset cardiomyopathy

Time frame: 3 years

Sponsors and collaborators

Lead sponsor

Washington University School of Medicine

Other

Collaborators

Boston Children's Hospital
Nationwide Children's Hospital
University of California, Davis
University of Minnesota

Registry information

Official study title

Clinical Outcomes Validation in Non Ambulatory and Young Boys/Men With Duchenne Muscular Dystrophy (DMD)

Acronym: DMD

Important dates

Study start: 2010
Primary completion: 2013
Study completion: 2013
First posted: Apr 5, 2010
Registry last updated: Jan 18, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed