Prednisone and other drugs in the corticosteroid family have become standard treatment for Duchenne muscular dystrophy (DMD) in much of the world for almost a decade. In this disorder, they slow the decline of muscle function and prolong walking ability. However, corticosteroids have significant side effects, such as weight gain, bone loss, cataracts and behavior changes.
Many questions about corticosteroid use in DMD remain, particularly about which medication, which dosage regimen, and what duration of treatment should be prescribed to maximize benefit and minimize harmful effects.
Now, with the support of the National Institute of Neurological Disorders and Stroke (NINDS), the first four sites in a 40-center, multinational trial exploring these questions have begun enrolling participants. More centers will open as they are ready.
300 boys on three regimens
In the phase 3 trial, investigators at medical centers in the United States, Canada, the United Kingdom, Germany and Italy will study young boys with DMD for five years.
Each participant will be assigned to receive one of three corticosteroid regimens for three to five years:
Prospective participants must:
Prospective participants must not:
The principal investigators are neurologist Robert Griggs at the University of Rochester Medical Center in New York and geneticist Kate Bushby at the Institute of Human Genetics in Newcastle Upon Tyne in the United Kingdom.
For details, including contact information and recruitment status (open or not yet open) for the study sites, see Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD) (or enter NCT01603407 in the search box at ClinicalTrials.gov).
For general information about U.S. sites, contact Kimberly Hart, U.S. project manager for FOR-DMD in Rochester, N.Y., at (585) 275-3767 or firstname.lastname@example.org.
The four sites that are currently open to recruitment are in Los Angeles; Rochester, N.Y.; Calgary, Alberta, Canada; and Newcastle Upon Tyne, United Kingdom.
Site-specific study coordinators at open sites follow.
University of California,Los Angeles
University of Rochester Medical Center
Alberta Children's Hospital
Institute of Human Genetics
International Centre for Life
Newcastle Upon Tyne
0191 241 8649
For more information
To learn more about corticosteroids and DMD, see: