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
This trial accepts healthy volunteersQualifiers
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 population
Subjects are recruited from across the country. Investigators have a website (www.imagingNMD.org) and advertise the study nationally through list serves. The study will be advertised at the website of non-profit MD organizations. General information will be emailed to faculty and colleagues around the country. Fliers and brochures will be distributed in participating local clinics, MDA clinics, non-profit organizations, local neuromuscular clinics, and in strategic locations in associated hospitals. Age-matched healthy men will be recruited to match subjects with Becker MD from local and university communities. Individuals interested in this study are asked to contact the site clinical coordinator, who will complete a telephone screening interview to assess eligibility.
Trial design
Design model
Case-control
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
550 Participants
are grouped into 3 trial groupsGroup A: Age Matched Controls
Group B: Boys/Men with DMD
Group C: Adults with Becker MD
Trial outcomes
Primary outcomes
1
Change from baseline in intramuscular lipid up to 3-10 years
In BMD and DMD, the from baseline in intramuscular lipid of upper/ lower extremity and trunk/respiratory muscles, as well as composite measures. MR measures of intramuscular lipid will be measured in yearly intervals for a period up to 3-10 years.
Time frame
Change in baseline up to 3-10 years2
Change from baseline in muscle T2 up to 3 months in DMD
In a subgroup of subjects the effect of corticosteroids on muscle T2 will be measured at 3 and 6 months. Muscle T2 is a noninvasive marker of muscle damage/inflammation and will be measured using MR. This substudy requires its own Primary and Secondary Outcome measures.
Time frame
Change in baseline up to 3 months3
Correlation between MR measures of intramuscular lipid, functional endpoints and histological markers.
In both BMD and DMD, the correlation between MR measures and functional endpoints will be determined, as well as the ability of MR measures to predict future change and loss in function.
Time frame
Through study completion, an average of 1 yearSecondary outcomes
1
Change from baseline in muscle T2 up to 5-10 years
Muscle T2 will be measured in the lower extremity and/or upper extremity muscles using MR at yearly intervals up to 5-10 years. We will report the change for each year interval.
Time frame
Change in baseline up to 5-10 years2
Change from baseline in muscle contractile area up to 5-10 years
Muscle contractile area will be measured in the lower extremity and/or upper extremity muscles using MR at yearly intervals up to 5 years. We will report the change for each year interval.
Time frame
change in baseline up to 5-10 years3
Change from baseline in muscle T2 at 6 months
In a subgroup of subjects the effect of corticosteroids on muscle T2 will be measured at 3 and 6 months. Muscle T2 is a noninvasive marker of muscle damage/inflammation and will be measured using MR. This substudy requires its own Primary and Secondary Outcome measures.
Time frame
Change in baseline up to 6 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
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
Source ClinicalTrials.gov