Status:
RECRUITING
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Lead Sponsor:
University of Florida
Collaborating Sponsors:
Oregon Health and Science University
Children's Hospital of Philadelphia
Conditions:
Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Eligibility:
MALE
5-62 years
Brief Summary
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...
Detailed Description
The overall objective of this proposal is to validate the potential of noninvasive magnetic resonance imaging (MRI) and spectroscopy (MRS) to monitor disease progression and to serve as an outcome mea...
Eligibility Criteria
Inclusion
- Inclusion Criteria for boys with DMD:
- 1\. 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
- Inclusion Criteria for adults with Becker MD:
- 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:
- 1\. Ambulatory males (ages 18-62) without disease or injury to the lower and/or upper extremities will be eligible to participate in this study
- Exclusion Criteria:
- 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
Exclusion
Key Trial Info
Start Date :
September 1 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
August 31 2026
Estimated Enrollment :
550 Patients enrolled
Trial Details
Trial ID
NCT01484678
Start Date
September 1 2020
End Date
August 31 2026
Last Update
October 15 2025
Active Locations (3)
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1
University of Florida
Gainesville, Florida, United States, 32610
2
Oregon Health and Science University
Portland, Oregon, United States, 97239
3
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104