Robert Griggs, a professor of neurology at the University of Rochester (N.Y.), has received an MDA grant of $237,316 over three years to support travel costs for North American participants in a large, multinational trial to determine which corticosteroid treatment regimen is best for children with Duchenne muscular dystrophy (DMD).
The corticosteroid drug prednisone has been found to prolong strength in boys with DMD, and another corticosteroid, deflazacort, may also be beneficial.
A few different corticosteroid treatment schedules have been used in DMD in an attempt to find the right balance between the benefits of corticosteroids and their notorious side effects, such as weight gain and bone loss. Which one is best, however, remains an open question.
About the corticosteroid trial
The new trial, which aims to add worldwide data to help settle the question of which corticosteroid regimen in DMD is optimal, is being sponsored by the U.S. National Institutes of Health (NIH) and is now open at most of its planned 39 locations in the United States, Canada, Germany, Italy and the United Kingdom.
Prospective participants must be 4 to 7 years old and meet other study criteria. (For details and updated information on which sites are recruiting new participants, see Finding the Optimum Regimen for Duchenne Muscular Dystrophy, or enter NCT01603407 into the search box at ClinicalTrials.gov.)
The investigators say the average family participating in this multiyear trial will need to make several overnight trips to a study site, possibly imposing a significant financial burden. The MDA grant for travel and lodging is meant to ease that burden.
More about corticosteroids in DMD
For more on corticosteroid treatment and research, read:
About Clinical Trials
A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.