Status:
COMPLETED
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborating Sponsors:
Ipsen
Charley's Fund
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
5+ years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
Detailed Description
Detailed Description: 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 signifi...
Eligibility Criteria
Inclusion
- DMD diagnosed with mutational testing and/or complete absence of dystrophin on muscle biopsy
- 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
- Current or prior treatment with growth hormone or IGF-1 therapy
- 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
Key Trial Info
Start Date :
November 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2013
Estimated Enrollment :
44 Patients enrolled
Trial Details
Trial ID
NCT01207908
Start Date
November 1 2010
End Date
June 1 2013
Last Update
January 20 2021
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229