What is the purpose of the study?
The purpose of this trial is to determine if weekend oral corticosteroids improve development in infants and young boys with Duchenne Muscular Dystrophy (DMD).
Who can participate in the study?
We will enroll male infants and young boys between the age of 1 month through the age of 30 months. Participants must have the genetic mutation known to be causative for Duchenne Muscular Dystrophy, an appropriate degree of weakness for their age, and creatine kinase greater than 20 times the upper limit of normal.
What will happen during the study?
There will be 3 study visits, which will include a baseline visit, a follow-up examination after 6 months, and a follow-up examination after 12 months. The predicted time to complete all procedures and questionnaires is 2-3 hours per visit.
Gross motor development, fine motor development, speech, language and social skills will be assessed at baseline, after six months of treatment, and after twelve months of treatment.
We will compare the development of infants and young boys with DMD who are treated orally with prednisolone (10 mg/kg/week given in equal doses over two days) to the development of untreated DMD infants and young boys who have just completed one-year follow-up using the same measures.
We will also your information to our list of patients who are interested in being contacted about upcoming trials for their specific diagnosis.
Who can I contact if I am interested or if I have any questions?
You can contact either of the following clinical research coordinators for this study: