Clinical trial NCT01207908: Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
Official title: IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Duchenne Muscular Dystrophy
Keywords: DMD
Duchenne Muscular Dystrophy
Insulin like growth factor
6 minute walk test
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
Collaborators: name: Ipsen
name: Charley's Fund
First posted: 2010-09-23
Enrollment: 44
Sex: MALE
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
timeFrame: 6 months
Secondary outcomes:
measure: Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
timeFrame: 6 months
measure: Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
timeFrame: 6 months
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Meilan Rutter, MD
role: PRINCIPAL_INVESTIGATOR
References: