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

Completed

• Conditions: Duchenne Muscular Dystrophy

• Registration Number: NCT01098708
• Lead Sponsor: Washington University School of Medicine

Brief 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.

Study Timeline

• Study Start: 2010-03
• Study First Submitted: 2010-04-01
• Study First Submitted QC: 2010-04-01
• Study First Posted: 2010-04-05
• Primary Completion: 2013-11
• Study Completion: 2013-11
• Status Verified: 2017-01
• Last Update Submitted: 2017-01-17
• Last Update Posted: 2017-01-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 50

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Dystrophin gene mutations that predispose to early onset cardiomyopathy	3 years

Trial Locations

Locations (1)

Washington University; 🇺🇸 St. Louis, Missouri, United States