Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2017-03-22
Target enrollment:
Participant gender:
Summary
While it has been known for many years that corticosteroid use benefits boys with Duchenne
Muscular dystrophy (DMD), most clinicians do not consider treating until after age 3 or 4
years of age. The primary reason for the delay is that daily corticosteroid use has many side
effects including short stature, obesity, and osteoporosis. A recent randomized blinded study
of weekend oral corticosteroid use over one year showed equal improvement in strength with
fewer side effects, particularly as related to growth and cushingoid changes. The
investigators will test the efficacy of oral weekend corticosteroid use in infants and young
boys with DMD who are under age 30 months. The investigators have demonstrated that the
Bayley-III Scales of Infant development shows that infants and young boys in this age group
who are untreated decline in abilities when compared to their peers. Here, in this Phase 2
historically controlled trial, the investigators will use these two measures and treat boys
at five Muscular Dystrophy Association-DMD centers
Phase:
Phase 2
Details
Lead Sponsor:
Washington University School of Medicine
Collaborators:
Feinberg School of Medicine, Northwestern University Nationwide Children's Hospital Nemours Hospital, Orlando, FL University of California, Davis University of Texas Southwestern Medical Center