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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00577577
• Date of registration: 18/12/2007
• Prospective Registration: No
• Primary sponsor: Insmed Incorporated
• Public title: 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
• Scientific 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
• Date of first enrolment: December 2007
• Target sample size: 60
• Recruitment status: Active, not recruiting
• URL: http://clinicaltrials.gov/show/NCT00577577
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Supportive Care
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Richard Moxley, M.D.
• Affiliation: University of Rochester Neuromuscular Disease Center

Key inclusion & 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



Age minimum: 21 Years
Age maximum: 65 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Myotonic Dystrophy Type 1

Intervention(s)

Drug: placebo

Drug: rhIGF-I/rhIGFBP-3

Primary Outcome(s)

Ambulation [Time Frame: Six Months]

Endurance [Time Frame: Six Months]

Cognitive function [Time Frame: Six months]

Quality of Life [Time Frame: Six Months]

Cholesterol and triglycerides [Time Frame: Six Months]

Muscle function and strength [Time Frame: Six months]

Safety and Tolerability [Time Frame: Six Months]

Insulin Resistance [Time Frame: Six Months]

Gastrointestinal function [Time Frame: Six Months]

Pain [Time Frame: Six Months]

Secondary ID(s)

INSM-110-1001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Muscular Dystrophy Association