Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
NCT ID: NCT01207908
Last Updated: 2021-01-20
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
44 participants
INTERVENTIONAL
2010-11-30
2013-06-30
Brief Summary
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Detailed Description
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DMD is a progressive degenerative muscle disorder for which there is no current cure. Glucocorticoids (GC) are often used to improve motor function and survival but have significant side effects such as growth failure, weight gain, insulin resistance and osteoporosis. IGF-1 stimulates both the proliferation and differentiation of skeletal muscle cells and is thus important for muscle repair and regeneration. IGF-1 offers potential as a therapeutic agent for DMD as it may improve or preserve motor function and reduce GC side effects such as growth failure and insulin resistance.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
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IGF-1
IGF-1 plus standard steroid treatment
IGF-1
IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
Standard steroid treatment alone
Standard daily steroid treatment for DMD
No interventions assigned to this group
Interventions
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IGF-1
IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps)
* Male
* Age \> 5 years of age
* Bone maturation (assess by bone age x-ray): \</= 11 years of age
* Daily GC (prednisone or deflazacort) therapy for \> 12 months
* Ambulatory
* Informed consent
* Willingness and ability to comply with all protocol requirements and procedures
Exclusion Criteria
* Non-ambulatory
* Pubertal (based on clinical Tanner staging examination)
* Congestive cardiac failure
* History of intracranial hypertension
* Daytime ventilatory dependence (non-invasive or tracheostomy)
* Concomitant therapy - any other medications/supplements that would be considered, in the opinion of the investigators, to affect muscle function, need to have been started 3 months prior to enrollment
* Patients enrolled in other clinical drug trials
* Any physical or mental conditions which may, in the investigators'opinions, render the subject unable to complete the tasks of the study appropriately
* There will be no selection by ethnicity
5 Years
MALE
No
Sponsors
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Ipsen
INDUSTRY
Charley's Fund
OTHER
Children's Hospital Medical Center, Cincinnati
OTHER
Responsible Party
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Principal Investigators
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Meilan Rutter, MD
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Medical Center, Cincinnati
Locations
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Countries
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Related Links
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Genetics Home Reference
Duchenne and Becker Muscular Dystrophy
Muscular Dystrophy
Other Identifiers
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2010-1491
Identifier Type: -
Identifier Source: org_study_id
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