Your guide to Nationwide Children's
myChildren's provides access to our main campus and offsite locations, wayfinding, physician and service information and more. Available for iPhones and Android.
Purpose of Study
The Newcastle University and the University of Rochester, in collaboration with the United States National Institutes of Health, and the National Institute of Neurological Disorders and Stroke, are conducting a phase III study with corticosteroids in boys with Duchenne Muscular Dystrophy (FOR DMD study).
Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects. Currently, different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all.
The FOR DMD study aims to compare three ways of giving corticosteroids to boys with DMD to determine which increases muscle strength the most, and which causes the fewest side effects.
Using the results of this study, we aim to provide patients and families with clearer information about the best way to take these drugs.
This study will look at three ways of taking corticosteroids by the mouth:
Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment
All three dosages are commonly used in boys with DMD and have shown to be beneficial.
In this study there is no placebo group, which means that all participants will receive active drugs (Prednisone or Deflazacort). However, neither the boys nor the clinicians will know which treatment or regime the boy is taking.
The study will recruit 300 boys around Europe, United States and Canada.
In North America, 16 centers will take part in the study:
Alberta Children's Hospital Penn State Children's Hospital
Children's Memorial Hospital, Chicago SUNY Downstate Med Center
Health Sciences Centre Winnipeg University of California, Davis
Kansas University Medical Center University of California Los Angeles
Kennedy Krieger Institute University of New Mexico
London Health Sciences Center University of Rochester Medical Center
Nemours Children’s Hospital, Orlando University of Texas Southwestern Medical Center
Nationwide Children’s Hospital Vanderbilt Children's Hospital
Patients who do not attend one of these hospitals for their routine follow-up can also participate, but will have to travel to their closest participating site to receive the study drug and for the check-ups.
Participants will receive study medication for a minimum of three years and a maximum of five years (depending on how early the boy was recruited into the study) and participation involves visits to the study hospital every three months for the first 6 months and every six months thereafter. At these visits we will be repeating many of the tests your child usually has in clinic for his routine DMD follow up.
Who can participate:
In order to take part in the study boys need to fulfil a number of criteria. These can only be checked when you come into the clinic. However, at this stage if your child may be eligible if he
Has a genetically confirmed diagnosis of DMD
Is aged 4-7 years (before 8th birthday)
Has never taken steroids (by mouth)
Who to contact:
If you feel that your child might be able to participate in this trial, please feel free to discuss it with your doctor locally. Alternatively, if you would like further information, please contact the University of Rochester Medical Center: Kim Hart | Phone: 1 (585) 275-3767.