Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

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.

Inclusion criteria

• DMD diagnosed with mutational testing and/or complete absence of dystrophin on muscle biopsy
• Proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps)
• Male
• Age > 5 years of age
• Bone maturation (assess by bone age x-ray): </= 11 years of age
• Daily GC (prednisone or deflazacort) therapy for > 12 months
• Ambulatory
• Informed consent
• Willingness and ability to comply with all protocol requirements and procedures

Exclusion criteria

• Current or prior treatment with growth hormone or IGF-1 therapy
• Non-ambulatory
• Pubertal (based on clinical Tanner staging examination)
• Congestive cardiac failure
• History of intracranial hypertension
• Daytime ventilatory dependence (non-invasive or tracheostomy)
• Concomitant therapy - any other medications/supplements that would be considered, in the opinion of the investigators, to affect muscle function, need to have been started 3 months prior to enrollment
• Patients enrolled in other clinical drug trials
• Any physical or mental conditions which may, in the investigators'opinions, render the subject unable to complete the tasks of the study appropriately
• There will be no selection by ethnicity