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

• Status: Unknown status
• Trial end date: 2009-03-01
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Insmed Incorporated
• Collaborator: Muscular Dystrophy Association
• Treatments: Insulin; Insulin, Globin Zinc; Mitogens

Criteria

Inclusion Criteria (list is not inclusive):

• A diagnosis of DM1, confirmed by DM1 genetic mutation

• 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

Exclusion Criteria (list is not inclusive):

• Congenital DM1

• Weight greater than 100 kg or body mass index greater than 30 kg/m2

• Prior treatment with glucocorticoids, anabolic steroids, testosterone, growth hormone, investigational agent within 60 days of screening

• Current diagnosis or history of malignancy expect for surgically cured skin cancer or pilomatricoma

• Changes in lipid lowering medications during the 3 months prior to screening

• Diaphragmatic weakness such that patients are unable to tolerate the supine position, or swallowing impairment such that patients are unable to maintain nutrition without use of gastrostomy.

• Major psychiatric illness (major depression, bipolar disorder or schizophrenia) within twelve months of screening

• History of non-compliance with other therapies