This trial is active, not recruiting.

Condition duchenne muscular dystrophy
Treatments igf-1, standard steroid treatment
Phase phase 1/phase 2
Sponsor Children's Hospital Medical Center, Cincinnati
Collaborator Tercica- Subsidiary of Ipsen
Start date November 2010
End date October 2012
Trial size 40 participants
Trial identifier NCT01207908, 2010-1491


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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking single blind (outcomes assessor)
Primary purpose treatment
IGF-1 plus standard steroid treatment
igf-1 Increlex (mecasermin [rDNA origin] injection)
IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
(No Intervention)
standard steroid treatment
Will complete all study related procedures and evaluations

Primary Outcomes

Six Minute Walk Test (6MWT)
time frame: 6 months

Secondary Outcomes

Growth Rate
time frame: 6 months
Difference in motor function
time frame: 6 months
Safety parameters
time frame: 6 months

Eligibility Criteria

Male participants at least 5 years old.

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): 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

Additional Information

Official title IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy
Principal investigator Meilan Rutter, MD
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.
Trial information was received from ClinicalTrials.gov and was last updated in June 2012.
Information provided to ClinicalTrials.gov by Children's Hospital Medical Center, Cincinnati.