NCT01603407

Finding the Optimum Regimen for Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 196 (actual)
• Sponsor: University of Rochester · Academic / Other
• Sex: Male
• Age: 4 Years – 7 Years
• Healthy volunteers: Not accepted

Summary

The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.

Detailed description

Boys with Duchenne muscular dystrophy experience progressive muscle weakness as they grow up. Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Benefits include an increase in the length of time that boys could continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possible protection against the development of heart problems. Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects of the drug. No controlled, long-term study has ever looked at the effects of different corticosteroids to see which one improves strength the most and which one causes the fewest side effects, over a period of time. Different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all. The FOR DMD study will enroll boys with DMD ages 4-7. The study will look at three ways of taking the following corticosteroids by the mouth to determine which increases muscle strength the most, and which causes the fewest side effects: 1. Prednisone 0.75mg/kg/day 2. Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment 3. Deflazacort 0.9mg/kg/day. The study will take place at 40 academic medical centers in the United States, Canada, United Kingdom, Germany and Italy.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Prednisone	daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
DRUG	Prednisone	intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
DRUG	Deflazacort	daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months

Timeline

• Start date: 2013-01-01
• Primary completion: 2019-11-01
• Completion: 2019-11-01
• First posted: 2012-05-23
• Last updated: 2022-08-12
• Results posted: 2022-08-12

Locations

32 sites across 5 countries: United States, Canada, Germany, Italy, United Kingdom