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
- A diagnosis of DM1, confirmed by DM1 genetic mutation
- Age 21 to 65 years (inclusive)
- Ability to walk 30 feet - assistance with cane and/or leg bracing permitted
- 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