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

Insmed

Status and phase: Completed; Phase 2

Conditions: Myotonic Dystrophy Type 1

Treatments: Drug: placebo; Drug: rhIGF-I/rhIGFBP-3

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00577577

INSM-110-1001

Details and patient eligibility

About

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

Full 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

Enrollment: 69 patients

Sex: All

Ages: 21 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Supportive Care

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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69 participants in 2 patient groups, including a placebo group

rhIGF-I/rhIGFBP-3

Experimental group

Treatment:

Drug: rhIGF-I/rhIGFBP-3

Placebo

Placebo Comparator group

Treatment:

Drug: placebo