A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL EVALUATING THE EFFICACY AND SAFETY OF HUMAN ALLOGENEIC CARDIOSPHERE-DERIVED
CELLS FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (HOPE-3)
Patients must meet the following criteria for entry in the study:
- Male subjects at least 10 years of age.
- Diagnosis of DMD based on clinical and phenotypic manifestations consistent with DMD (e.g., family history of DMD, elevated creatine kinase, dystrophin muscle biopsy, calf pseudohypertrophy, history of Gowers’ sign, and gait impairment before 7 years of age) as confirmed by the physician.
- Confirmatory genetic testing performed to have reached a diagnosis of DMD at any time in the past or currently performed at a Clinical Laboratory
- Treatment with a systemic glucocorticoid is required for at least 12 months