NCT01098708

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

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 50 (actual)
• Sponsor: Washington University School of Medicine · Academic / Other
• Sex: Male
• Age: 7 Years – 22 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Duchenne Muscular Dystrophy

Timeline

• Start date: 2010-03-01
• Primary completion: 2013-11-01
• Completion: 2013-11-01
• First posted: 2010-04-05
• Last updated: 2017-01-18

Locations

1 site across 1 country: United States