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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01098708

Acronym

DMD

Enrollment

Registered

2010-04-05

Start date

2010-03-31

Completion date

2013-11-30

Last updated

2017-01-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD, non ambulatory

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

MALE

Age

7 Years to 22 Years

Healthy volunteers

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. -

Design outcomes

Primary

Measure	Time frame
Dystrophin gene mutations that predispose to early onset cardiomyopathy	3 years

Countries

United States

Outcome results