The National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, is looking for individuals to participate in clinical studies. Participating in clinical trials allows you to play an active role in research on the nature and causes of many disorders of the brain and nervous system, and to possibly help physician-scientists develop future treatments. The information below is designed to help you quickly learn about actively recruiting research studies for which you or someone you know may be eligible.
This study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine how best to increase muscle strength with the fewest side effects.
Boys with DMD experience progressive muscle weakness as they grow up and corticosteroids currently are the only medicine that has been shown to increase their muscle strength. Benefits include an increase in the length of time that boys could continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possible protection against the development of heart problems.
Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects of the drug. No controlled, long-term study has ever looked at the effects of different corticosteroids to see which one improves strength the most and which one causes the fewest side effects, over a period of time. Different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all.
The FOR DMD study will look at three ways of taking the following corticosteroids by the mouth to determine which increases muscle strength the most, and which causes the fewest side effects: Prednisone 0.75mg/kg/day; Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment; and Deflazacort 0.9mg/kg/day. Participants may be able to take concurrent treatments during participation, because it is even more important to know the best corticosteroid for combination therapy.
The study will take place at 40 academic medical centers in the United States, Canada, United Kingdom, Germany, and Italy.
Multiple Locations Worldwide
Last Reviewed March 4, 2016