Overview

This trial is active, not recruiting.

Condition myotonic dystrophy type 1
Treatments rhigf-i/rhigfbp-3, placebo
Phase phase 2
Sponsor Insmed
Collaborator Muscular Dystrophy Association
Start date December 2007
End date March 2009
Trial size 60 participants
Trial identifier NCT00577577, INSM-110-1001

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

United States No locations recruiting
Other Countries No locations recruiting

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

Primary Outcomes

Measure
Endurance
time frame: Six Months
Ambulation
time frame: Six Months
Cognitive function
time frame: Six months
Insulin Resistance
time frame: Six Months
Cholesterol and triglycerides
time frame: Six Months
Muscle function and strength
time frame: Six months
Pain
time frame: Six Months
Gastrointestinal function
time frame: Six Months
Quality of Life
time frame: Six Months
Safety and Tolerability
time frame: Six Months

Eligibility Criteria

Male or female participants from 21 years up to 65 years old.

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

Additional Information

Official 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
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
Trial information was received from ClinicalTrials.gov and was last updated in July 2008.
Information provided to ClinicalTrials.gov by Insmed.