Brief Title
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
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 Phase
Phase 2
Study Type
Interventional
Primary Outcome
Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance
Condition
Myotonic Dystrophy Type 1
Intervention
rhIGF-I/rhIGFBP-3
Study Arms / Comparison Groups
rhIGF-I/rhIGFBP-3
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
69
Start Date
December 2007
Completion Date
December 29, 2008
Primary Completion Date
December 29, 2008
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
Gender
All
Ages
21 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Richard Moxley, M.D., ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00577577
Organization ID
INSM-110-1001
Responsible Party
Sponsor
Study Sponsor
Insmed Incorporated
Collaborators
Muscular Dystrophy Association
Study Sponsor
Richard Moxley, M.D., Study Chair, University of Rochester Neuromuscular Disease Center
Verification Date
December 2021