Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Description

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.

Conditions

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy

Study Overview

Study Details

Study overview

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.

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Condition
Duchenne Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Gainesville

University of Florida, Gainesville, Florida, United States, 32610

Portland

Oregon Health and Science University, Portland, Oregon, United States, 97239

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Males with a contraindication to an MR examination
  • 2. Males with unstable medical problems
  • 3. Males who are not able to cooperate during testing
  • 4. Males with a secondary condition that may impact muscle metabolism, muscle function or functional ability (i.e. cerebral palsy, endocrine disorders, mitochondrial disease)
  • 5. Daytime ventilation
  • 6. Implantable Cardioverter Defibrillator- (ICD) or pace maker
  • 7. Healthy boys/men who participate in competitive sports specific training in excess of 8 hours per week

Ages Eligible for Study

5 Years to 62 Years

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

Yes

Collaborators and Investigators

University of Florida,

William Rooney, PhD, PRINCIPAL_INVESTIGATOR, Oregan Health and Science University

H. Lee Sweeney, PhD, PRINCIPAL_INVESTIGATOR, University of Florida

Krista Vandenborne, PhD, PRINCIPAL_INVESTIGATOR, University of Florida

Study Record Dates

2025-08-31