Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
Completed · Phase 1/Phase 2
Conditions studied: Duchenne Muscular Dystrophy
In brief
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
Key facts
- Study ID
- NCT01207908
- Run by
- Children's Hospital Medical Center, Cincinnati
- People needed
- 44
- Starts
- 2010-11-01
- Expected to finish
- 2013-06-01
- Last updated by the study team
- 2021-01-20
Who can join
Age: 5 and older. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
Full record on ClinicalTrials.gov
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