Using MRI and biomarkers to monitor muscular dystrophy progression
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
This study is testing if MRI and other tests can help track how Duchenne and Becker muscular dystrophies change over time in boys and men, and see how these changes relate to their daily activities and treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 550 (estimated) |
| Ages | 5 Years to 62 Years |
| Sex | Male |
| Sponsor | University of Florida Academic / other |
| Locations | 3 sites (Gainesville, Florida and 2 other locations) |
| Trial ID | NCT01484678 on ClinicalTrials.gov |
What this trial studies
This research aims to evaluate the effectiveness of magnetic resonance imaging (MRI) and spectroscopy (MRS) in tracking the progression of Duchenne and Becker muscular dystrophies. The study will compare muscle conditions in boys and men with Duchenne muscular dystrophy (DMD) to healthy individuals over a period of 5-10 years, assessing muscle damage and fat content in relation to daily activity performance. Additionally, it will explore the effects of corticosteroid treatment on muscle measurements and map the progression of Becker muscular dystrophy in adults. The goal is to establish non-invasive biomarkers that can serve as reliable outcome measures for future clinical trials.
Who should consider this trial
Good fit: Ideal candidates include ambulatory and non-ambulatory males aged 5-30 with Duchenne muscular dystrophy and ambulatory males aged 18-62 with Becker muscular dystrophy.
Not a fit: Patients with co-existing conditions affecting muscle function or those outside the specified age ranges may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a non-invasive method to monitor disease progression in muscular dystrophy, improving clinical trial outcomes and patient care.
How similar studies have performed: Other studies have shown promise in using MRI and spectroscopy for monitoring muscular dystrophy, but this approach aims to validate these methods in a multi-centered setting.
Eligibility criteria
Show full inclusion / exclusion criteria
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: 1. Ambulatory males (ages 18-62) without disease or injury to the lower extremities 2. 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: 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
Where this trial is running
Gainesville, Florida and 2 other locations
- University of Florida — Gainesville, Florida, United States (Recruiting)
- Oregon Health and Science University — Portland, Oregon, United States (Recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Active_not_recruiting)
Study contacts
- Principal investigator: William Rooney, PhD — Oregan Health and Science University
- Study coordinator: Krista Vandenborne, PhD
- Email: kvandenb@phhp.ufl.edu
- Phone: 352-273-6100
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.