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completed

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

key information

study id #: NCT01098708

condition: Duchenne Muscular Dystrophy

status: completed

purpose:

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.

mechanism of action: No pharmaceutical intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT01098708

last updated: November 22, 2018

study details

start date: March 2010

estimated completion: November 2013

size / enrollment: 50

study 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.

primary outcomes:

  • Dystrophin gene mutations that predispose to early onset cardiomyopathy [ Time Frame: 3 years ]

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.

study contacts

sponsor: Washington University School of Medicine

investigators: Anne Connolly, M.D.

locations: United States