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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
History of Changes

December 18, 2007
July 21, 2008
December 2007
March 2009   (final data collection date for primary outcome measure)
  • 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 ]
Same as current
Complete list of historical versions of study NCT00577577 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
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
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

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

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
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Supportive Care
Myotonic Dystrophy Type 1
  • 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.
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
60
March 2009
March 2009   (final data collection date for primary outcome measure)

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
Both
21 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00577577
INSM-110-1001
Yes
Christy ONeal, Study Manager, Insmed Incorporated
Insmed
Muscular Dystrophy Association
Study Chair: Richard Moxley, M.D. University of Rochester Neuromuscular Disease Center
Insmed
July 2008

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP