Study of Osilodrostat with Glucocorticoid Replacement for Treating Endogenous Cushing Syndrome

What this Trial Is About

Researchers are studying patients with endogenous Cushing syndrome to understand how often adrenal insufficiency occurs when treated with osilodrostat combined with glucocorticoid replacement. The study also examines new steroid biomarkers to monitor osilodrostat dosing, and assesses treatment durability, safety, and clinical improvement. This observational study aims to provide important insights into managing this condition with a block-and-replace approach. The study has two phases. In Phase 1 Titration, participants start with a low dose of osilodrostat and add methylprednisolone replacement. Doses are adjusted to reach specific cortisol level goals, with frequent monitoring and communication. Once these goals are met, participants enter Phase 2 Maintenance, where they continue treatment for 48 weeks with periodic cortisol tests to track treatment effects. Participants will regularly provide blood and saliva samples for cortisol and steroid profiling, and report symptoms related to adrenal insufficiency. Researchers will track clinical measures such as blood pressure, weight, and quality of life over about 48 weeks. The main outcome is the percentage of participants experiencing adrenal insufficiency during Phase 1, along with various secondary health and hormone measures throughout the study.

A Block-and-Replace Therapy With Osilodrostat and Concomitant Glucocorticoid Replacement