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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
196
daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
Forced Vital Capacity
Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Rise From the Floor Velocity
Reciprocal of time to rise from the floor
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score
The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
North Star Ambulatory Assessment (NSAA) Score
The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects. The activities are graded as follows: 2 - "Normal" - no obvious modification of activity 1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
6 Minute Walk Test
Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
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University of California Los Angeles (UCLA) Medical Center
Los Angeles, California, United States
University of California Davis Medical Center
Sacramento, California, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Nemours Children's Hospital
Orlando, Florida, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of New Mexico Health Science Center
Albuquerque, New Mexico, United States
University of Rochester Medical Center
Rochester, New York, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
...and 22 more locations
Range of Motion (Goniometry) of Left Ankle
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Range of Motion (Goniometry) of Right Ankle
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Number of Participants Who Tolerated the Regimen
The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication.
Time frame: 3 years
Heart Rate
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time frame: 36 months
Quality of Life - Parent
Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child.
Time frame: Average of Months 12, 24, and 36 visits
Quality of Life- Child
Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life.
Time frame: Average of Months 12, 24, and 36 visits
Left Ventricular Ejection Fraction Percent
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time frame: 36 months
Fractional Shortening Percent
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time frame: 36 months
PR Interval
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time frame: 36 months
Participant Weight
Time frame: 36 months
Participant Height
Time frame: 36 months
Participant Body Mass Index
Time frame: 36 months