Clinical Trial: Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional

Official Title: A Placebo Controlled, Randomized, Double-Blind Phase II Clinical Trial to Evaluate Tolerability, Safety and Efficacy Endpoints After Administration of Recombinant Human Insulin-Like Growth Factor-I/Re

Brief Summary: To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance, ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and strength, pain, gastrointestinal functioning, and quality of life endpoints in DM1 patients

Detailed Summary:

Efficacy Measures:

Endurance, Ambulation, Cognitive function, Insulin resistance, Cholesterol and triglycerides, Muscle function and strength, Pain, Gastrointestinal function, Quality of life

MINIMUM INCLUSION CRITERIA

  1. A diagnosis of DM1, confirmed by DM1 genetic mutation
  2. Age 21 to 65 years (inclusive)
  3. Ability to walk 30 feet - assistance with cane and/or leg bracing permitted
  4. Able to self-administer study medication by subcutaneous injection or caregiver is available to administer study medication

Sponsor: Insmed Incorporated

Current Primary Outcome:

  • Endurance [ Time Frame: Six Months ]
  • Ambulation [ Time Frame: Six Months ]
  • Cognitive function [ Time Frame: Six months ]
  • Insulin Resistance [ Time Frame: Six Months ]
  • Cholesterol and triglycerides [ Time Frame: Six Months ]
  • Muscle function and strength [ Time Frame: Six months ]
  • Pain [ Time Frame: Six Months ]
  • Gastrointestinal function [ Time Frame: Six Months ]
  • Quality of Life [ Time Frame: Six Months ]
  • Safety and Tolerability [ Time Frame: Six Months ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Insmed Incorporated

Dates:
Date Received: December 18, 2007
Date Started: December 2007
Date Completion: March 2009
Last Updated: July 21, 2008
Last Verified: July 2008