The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
Detailed Description: DMD is a progressive degenerative muscle disorder for which there is no current cure. Glucocorticoids (GC) are often used to improve motor function and survival but have significant side effects such as growth failure, weight gain, insulin resistance and osteoporosis. IGF-1 stimulates both the proliferation and differentiation of skeletal muscle cells and is thus important for muscle repair and regeneration. IGF-1 offers potential as a therapeutic agent for DMD as it may improve or preserve motor function and reduce GC side effects such as growth failure and insulin resistance.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
44
IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Outcome Measure Data Table shows change of 6-Minute Walk Distance at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
Time frame: 6 months
Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Outcome Measure Data Table shows change of height velocity at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
Time frame: 6 months
Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Outcome Measure Data Table shows change of North Star Ambulatory Assessment (NSAA) score at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1). The NSAA is a 17-item scale that grades performance of various functional skills on a scale from 0 (unable), 1 (complete independently but with modifications), and 2 (complete without compensation). The range of NSAA score is from 0 to 34. The higher score indicates better motor function.
Time frame: 6 months
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