Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): A double-blind randomised placebo-controlled phase 3 trial

This was a randomized, double-blind trial that evaluated the efficacy of idebenone versus placebo in 65 patients with Duchenne muscular dystrophy who were not taking glucocorticoids. The primary endpoint was the change in spirometer-measured PEF % predicted; secondary endpoints included changes in hospital-evaluated PEF, FVC, FEV1, MIP, MEP, and PCF. Hospital-based lung function testing was evaluated using a Vitalograph Pneumotrac spirometer; additional home-based testing was undertaken using a Vitalograph Asma-1 device.