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

Study Status: Not yet recruiting
Recruit Status: Unknown status
Study Type: Interventional

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

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 Summary:

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.


Sponsor: Children's Hospital Medical Center, Cincinnati

Current Primary Outcome: 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.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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.


Original Secondary Outcome: Same as current

Information By: Children's Hospital Medical Center, Cincinnati

Dates:
Date Received: September 22, 2010
Date Started: November 2010
Date Completion: June 2013
Last Updated: June 12, 2012
Last Verified: June 2012