Overview
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
Status:
Completed
Completed
Trial end date:
2013-06-01
2013-06-01
Target enrollment:
0
0
Participant gender:
Male
Male
Summary
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).Phase:
Phase 1/Phase 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Children's Hospital Medical Center, CincinnatiCollaborators:
Charley's FundIpsen
Treatments:
MecaserminCriteria
Inclusion Criteria:- 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):
- 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:
- 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