Conditions: Myotonic Dystrophy Type 1
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 69
Healthy volunteers: no

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 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

Arms:
arm 1: None arm 2: None

Interventions: intervention 1: rhIGF-I/rhIGFBP-3 intervention 2: placebo
Intervention descriptions: intervention 1: 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. intervention 2: 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.

Protocol document: