What this study is
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.
Study details
- Sponsor
- University of Florida
- Conditions
- Duchenne Muscular Dystrophy · Becker Muscular Dystrophy
- Enrolling
- 550 people
- Starts
- 2020-09-01
- Est. completion
- 2026-08-31
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 5 Years – 62 YearsSex MALEHealthy volunteers Acceptedboys with DMD
You may qualify if
- Ambulatory and non-ambulatory males (ages 5-30 at baseline testing) previously diagnosed with DMD based on:; 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
You can't join if
- 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)
- Daytime ventilation
- Implantable Cardioverter Defibrillator- (ICD) or pace maker
- Healthy boys/men who participate in competitive sports specific training in excess of 8 hours per week
adults with Becker MD
You may qualify if
- Ambulatory males (ages 18-62) without disease or injury to the lower extremities
- Specific recruitment of a subset of individuals with deletion mutations in the dystrophin gene involving either exon 51 or exon 45. Inclusion Criteria for age matched controls for Becker MD subjects:
- Ambulatory males (ages 18-62) without disease or injury to the lower and/or upper extremities will be eligible to participate in this study
You can't join if
- 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)
- Daytime ventilation
- Implantable Cardioverter Defibrillator- (ICD) or pace maker
- Healthy boys/men who participate in competitive sports specific training in excess of 8 hours per week
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (3)
2 openUniversity of Florida
Gainesville, Florida, United States
- Krista Vandenborne, PhD · Contact352-273-6100kvandenb@phhp.ufl.edu
- Kelly Rock, PhD · Contact352-294-5798k.rock@phhp.ufl.edu
- Krista Vandenborne, PhD · Principal investigator
RecruitingOregon Health and Science University
Portland, Oregon, United States
- William Rooney, PhD · Contact503-418-1532rooneyw@ohsu.edu
- Olivia Huerta · Contact971-347-6084huertao@ohsu.edu
- Bill Rooney, PhD · Principal investigator
RecruitingChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Active, not recruiting
Study team
Sponsor
University of Florida
Study ID IRB201700056-N
Principal investigator
William Rooney, PhD
Oregan Health and Science University
Principal investigator
H. Lee Sweeney, PhD
University of Florida
Principal investigator
Krista Vandenborne, PhD
University of Florida
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.