Phase-2 Trial of 5mg/kg/Week Prednisolone in Young Boys With DMD
Anne M. Connolly
Summary
The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.
Description
The investigators know that the muscle destruction from Duchenne muscular dystrophy (DMD) begins in infancy and we previously demonstrated that motor decline in infancy compared to typically developing infants reflects that destruction. Because of the known side effects of daily corticosteroids, most physicians do not begin treatment until age 3-5 years. Most side effects (Cushingoid faces, linear growth arrest, and bone density loss) did not happen in infants and in ambulatory boys in two separate studies. However, in infants and young boys taking (10mg/kg/week), 56% of infants and young boys…
Eligibility
- Age range
- 0–2 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: * Subjects ages 1 month through 30 months * Weakness consistent with Duchenne on exam, creatine kinase ≥ 20 times the upper limit of normal, and genetic mutation known to be causative for DMD. Exclusion Criteria: * Prior treatment with Glucocorticosteroids
Interventions
- DrugPrednisolone
Liquid, 5mg/kg per week, for one year
Locations (4)
- Lurie Children's Hospital of ChicagoChicago, Illinois
- University of CincinnatiCincinnati, Ohio
- Nationwide Children's HospitalColumbus, Ohio
- University of Texas SouthwesternDallas, Texas