Clinical Trial: Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Effects of SomatoKine (Iplex) (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)

Brief Summary: The aim of this study is to investigate the safety and feasibility of daily subcutaneous injections of recombinant IGF1 complexed with IGF binding protein 3 (SomatoKine-INSMED) as a treatment for muscle wasting and weakness in myotonic dystrophy type 1.

Detailed Summary:
Sponsor: University of Rochester

Current Primary Outcome: The Number of Study Participants Who Safely Tolerated Somatokine [ Time Frame: 24 weeks ]

Safety and tolerability was measured via interval laboratory studies,electrocardiograms, echocardiograms, ultrasounds of the abdomen and pelvis, dual energy x-ray absorptiometry (DEXA) studies, chest and neck x-rays, and serial physical examinations. The participants had six inpatient evaluations at the University of Rochester General Clinical Research Center (Weeks 0, 8, 16, 24, 28, and 40) and nine outpatient evaluations. Patients also completed side effects diaries to record any adverse events in the interval time between inpatient and outpatient evaluations.


Original Primary Outcome: Safety and tolerability of SomatoKine during this study is the primary outcome measure in patients with DM-1.

Current Secondary Outcome:

Original Secondary Outcome:

Information By: University of Rochester

Dates:
Date Received: October 3, 2005
Date Started: November 2005
Date Completion:
Last Updated: June 20, 2012
Last Verified: June 2012