Osilodrostat is a promising new treatment option for patients with Cushing’s disease, and is generally well-tolerated, according to a clinical trial reported in Lancet Diabetes & Endocrinology.
This prospective, multicentre, open-label study included a double-blind, randomised withdrawal phase after a 24-week, open-label, single-arm treatment period. Patients aged 18–75 years with confirmed persistent or recurrent Cushing’s disease were enrolled (n=137) to receive osilodrostat (2-30 mg twice a day) or placebo at the end of the withdrawal period.
The primary endpoint was the proportion of participants who had been randomly assigned to treatment or placebo with a complete response at the end of the randomised withdrawal period. The key secondary endpoint was the proportion of participants with a complete response at the end of the single-arm, open-label period.
The results showed that a significantly higher proportion of patients in the osilodrostat group maintained a mean 24-h urinary free cortisol (UFC) concentration less than or equal to the upper limit of normal than the placebo group after randomised withdrawal. Two-thirds of patients had normalised mean 24-h UFC concentrations with osliodrostat by the end of the study. Osilodrostat was shown to be generally well-tolerated, with no unexpected side effects.