Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2013-06-01
• Target enrollment: 0
• Participant gender: 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 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborators: Charley's Fund; Ipsen
• Treatments: Mecasermin

Criteria

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