Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
|ClinicalTrials.gov Identifier: NCT01207908|
Recruitment Status : Unknown
Verified June 2012 by Children's Hospital Medical Center, Cincinnati.
Recruitment status was: Active, not recruiting
First Posted : September 23, 2010
Last Update Posted : June 13, 2012
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: IGF-1 Other: Standard steroid treatment||Phase 1 Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Single (Outcomes Assessor)|
|Official Title:||IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy|
|Study Start Date :||November 2010|
|Estimated Primary Completion Date :||October 2012|
|Estimated Study Completion Date :||June 2013|
IGF-1 plus standard steroid treatment
IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
Other Name: Increlex (mecasermin [rDNA origin] injection)
|No Intervention: Standard steroid treatment alone||
Other: Standard steroid treatment
Will complete all study related procedures and evaluations
- Six Minute Walk Test (6MWT) [ Time Frame: 6 months ]IGF-1 treatment motor function assessment by the difference in the distance traveled using the 6MWT.
- Growth Rate [ Time Frame: 6 months ]
- Difference in motor function [ Time Frame: 6 months ]Assessed by North Star Ambulatory Assessment, timed functional tests
- Safety parameters [ Time Frame: 6 months ]Measuring pulmonary function, cardiac function, and safety labs.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01207908
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|Principal Investigator:||Meilan Rutter, MD||Children's Hospital Medical Center, Cincinnati|