Researchers are looking for boys with Duchenne muscular dystrophy (DMD) to participate in the ongoing phase 3 clinical trial, Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD). The trial will compare three different corticosteroid regimens in boys with DMD, ages 3 to 7 years.
Corticosteroids, such as prednisone and deflazacort, work as anti-inflammatories or immunosuppressants and have been shown in clinical trials to stabilize or improve muscle function and strength in boys with DMD. Benefits often include prolonged ability to walk, increased respiratory function, decreased incidence of spinal surgery and stabilized cardiac function. Among the frequent side effects of steroid treatment are weight gain, osteoporosis, fat redistribution, growth slowing, cataracts, and behavioral and mood changes.
While prednisone has been approved by the U.S. Food and Drug Administration for use in DMD, deflazacort currently is the subject of an open-label expanded access program and is considered an investigational therapy.
The FOR-DMD trial aims to determine which of three corticosteroid treatment regimens increases muscle strength the most, and which causes the fewest side effects. Study results are expected to provide patients and families with clearer information about the best way to take these drugs.
The study, which expects to enroll 225 participants, is currently enrolling at more than 35 trial sites in the United States, Canada, Germany, Italy and the United Kingdom. The three treatment regimens are:
• Daily prednisone tablets – 0.75 milligrams per kilogram per day for 36-60 months
• Intermittent prednisone tablets – 0.75 milligrams per kilogram per day, 10 days on followed by 10 days off, for 36 to 60 months
• Daily deflazacort tablets – 0.9 milligrams per kilogram per day for 36-60 months
Participants must have a genetically-confirmed diagnosis of DMD, be able to rise independently from the floor, and meet other eligibility criteria.
MDA has a long history of supporting research and clinical study into the effects of corticosteroids in DMD and related diseases, with studies to determine mechanism of action, drug effects, side effects and best dosing regimen. An MDA human clinical trial grant currently is providing funding support to cover travel expenses for participants in the FOR-DMD trial.
To learn more about this trial, including site locations and complete inclusion and exclusion criteria, visit ClinicalTrials.gov and enter NCT01603407 into the search box, or contact Kimberly Hart at (585) 275-3767 or Kim_Hart@urmc.rochester.edu.