Cushing’s syndrome is a condition that occurs when the body is exposed to high levels of the hormone cortisol. Common complications of the condition include weight gain and stretch marks. Levoketoconazole is a ketoconazole stereoisomer known to be effective in suppressing the development of Cushing’s syndrome. This study aims to assess the safety and efficacy of levoketoconazole in patients with endogenous Cushing’s syndrome.
This multicenter, non-randomized, single-arm, open-label, phase-3 trial included a total of 94 patients aged 18 years or more with confirmed Cushing’s syndrome. The patients were assigned to oral levoketoconazole (150 mg twice daily, followed by up to 600 mg daily after normalization). The primary outcome of the study was the normalization of urinary free cortisol (mUFC) levels.
Of 94 patients included in the study, 80 (85%) had pituitary Cushing’s syndrome, with mean mUFC level 671.4 nmol/24 h, 4.9 times higher than the upper limit of normal. A total of 77 patients advanced to the maintenance phase, and 62 (81%) of them had mUFC normalization. The most common adverse events were nausea (32%) and headache (28%).
The research concluded that the twice-daily oral levoketoconazole treatment was associated with improvements in mUFC levels in patients with endogenous Cushing’s syndrome.