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

Cincinnati Children's Hospital Medical Center

Status and phase

Completed

Phase 2

Phase 1

Conditions

Duchenne Muscular Dystrophy

Treatments

Drug: IGF-1

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT01207908

2010-1491

Details and patient eligibility

About

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

Full 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.

Enrollment

44 patients

Sex

Male

Ages

5+ years old

Volunteers

No Healthy Volunteers

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