Multinational Trial to Evaluate Steroids in DMD

Some 300 boys with Duchenne muscular dystrophy (DMD) will soon be invited to participate in a multinational, multiyear study of three corticosteroid medication regimens.

Although corticosteroids such as prednisone have been used for many years to slow the course of DMD, and have been recommended by the American Academy of Neurology since 2005, there remains much debate about which steroids to use, how much to give, on what schedule and for what length of time.

Set to launch in 2008, with funding from the U.S. National Institutes of Health, the trial is expected to encompass approximately 59 sites in 11 countries.

Kate Bushby, professor of neuromuscular genetics at the International Centre for Life in Newcastle Upon Tyne, United Kingdom, and Robert Griggs, professor of neurology at the University of Rochester (N.Y.) Medical Center, are the principal investigators.

The doctors will test three regimens, using prednisone and deflazacort, in boys 4 to 7 years old for at least three years. They’ll compare the children’s muscle and respiratory functions, and their parents’ satisfaction with the treatment.