Clinical trial NCT00577577: 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
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myotonic Dystrophy Type 1
Lead sponsor: Insmed Incorporated
Sponsor class: INDUSTRY
Collaborators: name: Muscular Dystrophy Association
First posted: 2007-12-20
Enrollment: 69
Sex: ALL
Minimum age (years): 21
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

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

Eligibility 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

Primary outcomes:
measure: Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance
timeFrame: Baseline and Week 24
measure: Change From Baseline in Daily Step Count
timeFrame: Baseline and Week 24
measure: Peak Activity Index: Change From Baseline in Number of Steps Walked Per Minute During the 30 Minute Period of Fastest Walking
timeFrame: Baseline and Week 24
measure: Sustained Activity Index: Change From Baseline in the Highest Number of Steps Walked Per Minute Over 20 Minutes of Activity
timeFrame: Baseline and Week 24
measure: Change From Baseline in the Percentage of Time That Participants Spent Inactive
timeFrame: Baseline and Week 24
measure: Change From Baseline in Time Taken for Participants to Ascend and Descend 4 Stairs
timeFrame: Baseline and Week 24
measure: Change From Baseline in Time Taken to Traverse 30 Feet
timeFrame: Baseline and Week 24
measure: Change From Baseline in Purdue Pegboard Test Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in Forced Vital Capacity (FVC) Volume While Sitting or Lying Down
timeFrame: Baseline and Week 24
measure: Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted While Sitting or Lying Down
timeFrame: Baseline and Week 24
measure: Change From Baseline in Manual Muscle Test (MMT) Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in Selective Reminding Test T-Scores (Total Word and Delayed Words)
timeFrame: Baseline and Week 24
measure: Change From Baseline in Selective Reminding Test Raw Scores (Cued Recall and Recognition)
timeFrame: Baseline and Week 24
measure: Change From Baseline in Rey Complex Figure (RCF) Test Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in Letter-Number Sequencing (LNS) Test Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in Trail Making Test (TMT) Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in the Stroop Color Word Test Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline to Week 24 Scores on the Beck Depression Inventory II (BDI-II) Questionnaire
timeFrame: Baseline and Week 24
measure: Change From Baseline in Average Fasting Glucose Concentration in the Blood
timeFrame: Baseline - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution; Week 24 - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution
measure: Change From Baseline in Average Fasting Insulin Concentration in the Blood
timeFrame: Baseline - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution; Week 24 - Pre-dose and 30, 60, 90 and 120 minutes post glucose solution
measure: Change From Baseline in Qualitative Insulin Sensitivity Check Index (QUICKI)
timeFrame: Baseline and Week 24
measure: Change From Baseline in Insulin Sensitivity Index-Matsuda (ISI-Matsuda)
timeFrame: Baseline and Week 24
measure: Change From Baseline in Total Blood Cholesterol Level
timeFrame: Baseline and Week 24
measure: Change From Baseline in Total Blood Low-density Lipoproteins (LDL) Level
timeFrame: Baseline and Week 24
measure: Change From Baseline in Total Blood High-density Lipoproteins (HDL) Level
timeFrame: Baseline and Week 24
measure: Change From Baseline in Total Blood Triglycerides Level
timeFrame: Baseline and Week 24
measure: Change From Baseline in Gastro-esophageal Reflux Disease (GERD) Symptom Frequency Questionnaire (GSFQ) Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in Gastrointestinal Symptom Rating Scale for Irritable Bowel Syndrome (GSRS-IBS) Questionnaire Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in Swallowing Disturbance Questionnaire (SDQ) Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in Short Form (36) (SF-36) Questionnaire Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in Brief Pain Inventory (BPI) Questionnaire - Severity Scores
timeFrame: Baseline and Week 24
measure: Change From Baseline in Brief Pain Inventory (BPI) Questionnaire - Interference Scores
timeFrame: Baseline and Week 24
Secondary outcomes:
Other outcomes:
Locations:
city: Orange
country: United States
facility: University of California Irvine Medical Center; MDA, ALS and Neuromuscular Center
state: California
city: Sacramento
country: United States
facility: University of California, Davis
state: California
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University Medical School
state: Missouri
city: Rochester
country: United States
facility: University of Rochester, Neuromuscular Disease Center
state: New York
city: Columbus
country: United States
facility: Ohio State University Medical Center
state: Ohio
city: Portland
country: United States
facility: Oregan Health and Science University
state: Oregon
city: Galveston
country: United States
facility: Universit of Texas Medical Branch
state: Texas
city: San Antonio
country: United States
facility: University of Texas Health Science Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
Study officials:
affiliation: University of Rochester Neuromuscular Disease Center
name: Richard Moxley, M.D.
role: STUDY_CHAIR
References: