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Trial record 27 of 52 for:    Myotonic Dystrophy

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

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ClinicalTrials.gov Identifier: NCT00577577
Recruitment Status : Unknown
Verified July 2008 by Insmed Incorporated.
Recruitment status was:  Active, not recruiting
First Posted : December 20, 2007
Last Update Posted : July 23, 2008
Sponsor:
Collaborator:
Muscular Dystrophy Association
Information provided by:
Insmed Incorporated

Tracking Information
First Submitted Date  ICMJE December 18, 2007
First Posted Date  ICMJE December 20, 2007
Last Update Posted Date July 23, 2008
Study Start Date  ICMJE December 2007
Estimated Primary Completion Date March 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 19, 2007)
  • 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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT00577577 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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  ICMJE 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
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
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Supportive Care
Condition  ICMJE Myotonic Dystrophy Type 1
Intervention  ICMJE
  • 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.
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: December 19, 2007)
60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2009
Estimated Primary Completion Date March 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 21 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00577577
Other Study ID Numbers  ICMJE INSM-110-1001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Christy ONeal, Study Manager, Insmed Incorporated
Study Sponsor  ICMJE Insmed Incorporated
Collaborators  ICMJE Muscular Dystrophy Association
Investigators  ICMJE
Study Chair: Richard Moxley, M.D. University of Rochester Neuromuscular Disease Center
PRS Account Insmed Incorporated
Verification Date July 2008

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