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 - Full Text View

Sponsors and Collaborators:	Insmed Muscular Dystrophy Association
Information provided by:	Insmed
ClinicalTrials.gov Identifier:	NCT00577577

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 II

Genetics Home Reference related topics: familial encephalopathy with neuroserpin inclusion bodies myotonic dystrophy potassium-aggravated myotonia

Drug Information available for: Insulin Insulin-like growth factor I Mecasermin rinfabate BaseLine

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Supportive Care, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study
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

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:

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

A diagnosis of DM1, confirmed by DM1 genetic mutation
Age 21 to 65 years (inclusive)
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

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

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

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

Study placed in the following topic categories:

Myotonic Disorders
Neurodegenerative Diseases
Dystrophia myotonica 1
Insulin
Myotonia atrophica
Muscular Dystrophies
Muscular Diseases
Heredodegenerative Disorders, Nervous System

Muscular Disorders, Atrophic
Musculoskeletal Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Myotonic Dystrophy
Atrophy
Muscular dystrophy

Additional relevant MeSH terms:

Hypoglycemic Agents
Physiological Effects of Drugs
Nervous System Diseases
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009