DMD Natural History Study Seeks Participants

MRI_scan_in_progress hpResearchers are looking for boys and men, ages 5 to 18 years, to participate in a natural history study that is designed to assess the potential of imaging techniques to monitor disease progression and serve as an outcome measure for clinical trials in Duchenne muscular dystrophy (DMD). Both healthy volunteers and individuals with DMD are invited to participate. Participation in other clinical trials does not preclude participation in this study, and both ambulatory and non-ambulatory volunteers are being sought.

The study, known as “ImagingDMD,” is designed to help researchers determine how Magnetic Resonance Imaging (MRI) can be used to improve the design of future drug trials. MRI is an imaging technique that allows doctors to look inside the body using a scanner that sends out a strong magnetic field and radio waves. The procedure is painless, noninvasive and does not use radiation.

Study investigators will compare the muscles of boys with DMD with the muscles of healthy children of the same age, and will monitor the progression of the disease in those with DMD. They will examine the relationship between muscle function and muscle composition, by comparing muscle function testing results with images taken by MRI.

Trial participants will visit a study site yearly or in six-month intervals over a period of five to 10 years. Each clinical visit will include MRI and muscle function testing. An MRI scanning period takes about 90 minutes, during which participants will lie on a padded table and watch a movie. An investigator will always be present and a parent will be allowed in the scan room during imaging.

Throughout the study, participants will continue to see their regular doctor for routine care.

Trial sites are located at the University of Florida in Gainesville, The Children’s Hospital of Philadelphia (CHOP, and Oregon Health and Science University (OHSU) in Portland. Travel and hotel accommodations costs associated with clinic visits will be covered.

MDA has previously has contributed to foundational work in the use of MRI in DMD and is supporting the ImagingDMD trial in part through a development grant to Rebecca Willcocks, adjunct research assistant scientist at the University of Florida.

For more information about this trial, including complete inclusion and exclusion criteria, please visit ClinicalTrials.gov and enter NCT01484678. If you or someone you know may be interested in participating in this trial, contact Dr. Claudia Senesac at (352) 273-6453, or csenesac@phhp.ufl.edu.