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

Completed · Phase 2

Conditions studied: Duchenne Muscular Dystrophy

In brief

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

Key facts

Study ID: NCT02167217
Run by: Washington University School of Medicine
People needed: 25
Starts: 2014-04-17
Expected to finish: 2017-03-22
Last updated by the study team: 2018-12-21

Who can join

Age: 0 and older, up to 3. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

Appropriate degree of weakness for age, creatine kinase greater than 20 times the upper limit of normal, and genetic mutation known to be causative for Duchenne muscular dystrophy .
Appropriate degree of weakness for age, creatine kinase greater than 20 times the upper limit of normal and genetic or biopsy confirmation of Duchenne muscular dystrophy in a primary relative (e.g. brother or maternal uncle).
De-identified, genetic studies will be reviewed by collaborator Kevin Flanigan, MD prior to enrollment of subjects.
Age at entry: one month through 30 months.

You may not qualify if…

Prior treatment with corticosteroids

Where it is running

University of California Davis — Sacramento, California, United States
Nemours Hospital — Orlando, Florida, United States
Laurie Children's Hospital Of Chicago — Chicago, Illinois, United States
Washington University in Saint Louis — St Louis, Missouri, United States
Research Institute Center for Gene Therapy at Nationwide Children's Hospital — Columbus, Ohio, United States
University of Texas South Western Medical Center of Dallas — Dallas, Texas, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.