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

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

Detailed Description
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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.

Official Title
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IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Drug: IGF-1


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 5 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: IGF-1<br>IGF-1 plus standard steroid treatment | Drug: IGF-1<br>* IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.<br>* Other names: Increlex (mecasermin [rDNA origin] injection);|
| No Intervention: Standard steroid treatment alone<br>Standard daily steroid treatment for DMD | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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). | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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). | 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. | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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DMD, Duchenne Muscular Dystrophy, Insulin like growth factor, 6 minute walk test