welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!

Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

key information

study id #: NCT02167217

condition: Duchenne Muscular Dystrophy

status: completed


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

intervention: Prednisolone

mechanism of action: Glucocorticoid to delay decline in muscle strength

results: https://clinicaltrials.gov/ct2/show/results/NCT02167217

last updated: November 14, 2020