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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified July 2008 by Insmed.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Muscular Dystrophy Association
Information provided by:
Insmed
ClinicalTrials.gov Identifier:
NCT00577577
First received: December 18, 2007
Last updated: July 21, 2008
Last verified: July 2008
  Purpose

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


Condition Intervention Phase
Myotonic Dystrophy Type 1
Drug: rhIGF-I/rhIGFBP-3
Drug: placebo
Phase 2

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
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

Resource links provided by NLM:


Further study details as provided by Insmed:

Primary Outcome Measures:
  • Endurance [ Time Frame: Six Months ] [ Designated as safety issue: No ]
  • Ambulation [ Time Frame: Six Months ] [ Designated as safety issue: No ]
  • Cognitive function [ Time Frame: Six months ] [ Designated as safety issue: No ]
  • Insulin Resistance [ Time Frame: Six Months ] [ Designated as safety issue: No ]
  • Cholesterol and triglycerides [ Time Frame: Six Months ] [ Designated as safety issue: No ]
  • Muscle function and strength [ Time Frame: Six months ] [ Designated as safety issue: No ]
  • Pain [ Time Frame: Six Months ] [ Designated as safety issue: No ]
  • Gastrointestinal function [ Time Frame: Six Months ] [ Designated as safety issue: No ]
  • Quality of Life [ Time Frame: Six Months ] [ Designated as safety issue: No ]
  • Safety and Tolerability [ Time Frame: Six Months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 60
Study Start Date: December 2007
Estimated Study Completion Date: March 2009
Estimated Primary Completion Date: March 2009 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: rhIGF-I/rhIGFBP-3
    1.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit.
    Drug: placebo
    1.0 mg/kg rhIGF-I/rhIGFBP-3 or placebo daily, subcutaneous injections from baseline through the last day of the end of study visit.
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

Ages Eligible for Study:   21 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00577577

Locations
United States, California
University of California Irvine Medical Center; MDA, ALS and Neuromuscular Center
Orange, California, United States, 92868
University of California, Davis
Sacramento, California, United States, 95817
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Washington University Medical School
St. Louis, Missouri, United States, 63110
United States, New York
University of Rochester, Neuromuscular Disease Center
Rochester, New York, United States, 14642
United States, Ohio
Ohio State University Medical Center
Columbus, Ohio, United States, 43210
United States, Oregon
Oregan Health and Science University
Portland, Oregon, United States, 97239
United States, Texas
Universit of Texas Medical Branch
Galveston, Texas, United States, 77555-0539
University of Texas Health Science Center
San Antonio, Texas, United States, 78229
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84112
Sponsors and Collaborators
Insmed
Muscular Dystrophy Association
Investigators
Study Chair: Richard Moxley, M.D. University of Rochester Neuromuscular Disease Center
  More Information

No publications provided

Responsible Party: Christy ONeal, Study Manager, Insmed Incorporated
ClinicalTrials.gov Identifier: NCT00577577     History of Changes
Other Study ID Numbers: INSM-110-1001
Study First Received: December 18, 2007
Last Updated: July 21, 2008
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Myotonic Disorders
Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Insulin, Globin Zinc
Insulin
Mitogens
Hypoglycemic Agents
Physiological Effects of Drugs
Pharmacologic Actions
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on October 19, 2014