NCT00577577

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

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
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 69 (actual)
• Sponsor: Insmed Incorporated · Industry
• Sex: All
• Age: 21 Years – 65 Years
• Healthy volunteers: Not accepted

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 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

Conditions

• Myotonic Dystrophy Type 1

Interventions

Type	Name	Description
DRUG	rhIGF-I/rhIGFBP-3	1.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit.
DRUG	placebo	1.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit.

Timeline

• Start date: 2007-12-01
• Primary completion: 2008-12-29
• Completion: 2008-12-29
• First posted: 2007-12-20
• Last updated: 2022-01-06
• Results posted: 2022-01-06

Locations

12 sites across 1 country: United States