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

Myotonic Dystrophy Type 1 Clinical Trial

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/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) for 24 Weeks in Adults With Myotonic Dystrophy Type 1

Status Completed

Clinical Trial 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

Clinical Trial Description

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 ;

Related Conditions & MeSH terms

• Myotonic Dystrophy
• Myotonic Dystrophy Type 1

• NCT number: NCT00577577
• Study type: Interventional
• Source: Insmed Incorporated
• Status: Completed
• Phase: Phase 2
• Start date: December 2007
• Completion date: December 29, 2008