Optimum Steroid Regimen for Boys with Duchenne Muscular Dystrophy
Project Description:
Duchenne Muscular Dystrophy is the most common childhood Muscular Dystrophy with a birth incidence worldwide of 1 in 3,500 live male births. The natural history of the disease is devastating and currently there is no curative treatment. Untreated boys with DMD become progressively weak during childhood and are no longer ambulatory at a mean age of 9 years. Confinement to a wheelchair is followed by the development of spinal curvature, respitory failure and cardiomyopathy. Without intervention, the mean age of death is 19 years old.
Corticosteroids are the only pharmacological interventions currently available that increase muscle strength in DMD. The use of corticosteriods was first suggested in 1974. Both prednisone/prednisolone and deflazacort have been shown to increase muscle strength. This study is an international, multi-center, randomized, double-blind controlled trial comparing three active corticosteriod regimens over three years of treatment. It is anticipated that it will take two years to enroll the 300 subjects. The primary outcome variable will be a three-dimensional outcome comprising muscle function, respiratory function and treatment.
Keyword(s):
Duchenne Muscular Dystrophy, Prednisone, Deflazacourt, RCT
Core Function(s):
Performing Research or Evaluation
Area of Emphasis
Health-Related Activities
Target Audience:
Children/Adolescents with Disabilities/SHCN
Unserved or Under-served Populations:
Specific Groups
Primary Target Audience Geographic Descriptor:
Mulit-County
COVID-19 Related Data:
N/A
