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

Phase 1

Completed

Brief Summary: The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).

Detailed Description: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
IGF-1	IGF-1	IGF-1 plus standard steroid treatment

Primary Outcome Measures

Name	Time	Method
Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline	6 months	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).

Secondary Outcome Measures

Name	Time	Method
Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline	6 months	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).
Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline	6 months	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.