Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexMale
Age5-62
SponsorUniversity of Florida

About this trial

The purpose of this research study is to determine the potential of magnetic resonance imaging, spectroscopy, and whole body imaging to monitor disease progression and to serve as an objective outcome measure for clinical trials in Muscular Dystrophy (MD).

The investigators will compare the muscles of ambulatory or non-ambulatory boys/men with DMD with muscles of healthy individuals of the same age and monitor disease progression in those with DMD over a 5-10 year period. The amount of muscle damage and fat that the investigators measure will also be related to performance in daily activities, such as walking and the loss of muscle strength. In a small group of subjects the investigators will also assess the effect of corticosteroid drugs on the muscle measurements.

Additionally, the investigators will map the progression of Becker MD following adults with this rare disease. The primary objective is to conduct a multi-centered study to validate the potential of non-invasive magnetic resonance imaging and magnetic resonance spectroscopy to monitor disease progression and to serve as a noninvasive surrogate outcome measure for clinical trials in DMD and BMD. The secondary objective is to characterize the progressive involvement of the lower extremity, upper extremity, trunk/respiratory muscles in boys/men with DMD and BMD guiding clinical trials.

Eligibility criteria

Qualifiers

clinical features with onset of symptoms before age five

elevated serum creatine kinase level or

absence of dystrophin expression, as determined by immunostain or western blot (<2%) and/or DNA confirmation of a dystrophin mutation *Subjects will not be excluded based on corticosteroid treatment or other clinical trials

Ambulatory males (ages 18-62) without disease or injury to the lower extremities

Disqualifiers

Males with a contraindication to an MR examination

Males with unstable medical problems

Males who are not able to cooperate during testing

Males with a secondary condition that may impact muscle metabolism, muscle function or functional ability (i.e. cerebral palsy, endocrine disorders, mitochondrial disease)

Trial design

Treatments tested in this trial

  • Not listed

Trial groups

550 Participants
are grouped into 3 trial groups

Sponsors and collaborators

University of Florida

Lead sponsor

Oregon Health and Science University

Collaborator

Children's Hospital of Philadelphia

Collaborator

Shriners Hospitals for Children

Collaborator

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Collaborator