Osilodrostat for Autonomous Cortisol Secretion

This trial tests the safety and effects of osilodrostat, a medication for individuals with mild autonomous cortisol secretion (MACS). The goal is to assess its impact on cortisol levels and identify any side effects. Suitable candidates for this trial include those diagnosed with MACS, particularly if they have obesity, high blood pressure, or diabetes. This study is specifically for patients at the Mayo Clinic. Participants must be prepared to take oral medication and adhere to the study plan. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of people.

You may need to stop certain medications to join the trial, especially if you are currently using oral glucocorticoids, opioids over 20 MME/day, or medications that could interact with the study drug. It's best to discuss your current medications with the study team to see if any changes are needed.

Research has shown that osilodrostat is generally safe for people. It is already approved for treating Cushing syndrome, a condition characterized by excessive cortisol. Studies indicate that the drug is well-tolerated over time. However, some patients might experience side effects such as muscle pain and tiredness.

Osilodrostat is unique because it specifically targets cortisol production by inhibiting the enzyme responsible for its synthesis, which could lead to more precise control over cortisol levels in patients with mild autonomous cortisol secretion (MACS). Unlike traditional treatments that might focus on managing symptoms or using more general approaches, Osilodrostat directly addresses the source of the problem. Researchers are excited about this treatment because it offers a more targeted way to manage cortisol levels, potentially leading to better outcomes with fewer side effects.

Research has shown that osilodrostat effectively lowers cortisol levels by blocking a substance involved in cortisol production. For individuals with conditions like Cushing's syndrome, it helps balance cortisol levels, potentially improving quality of life and sleep. In this trial, participants with mild autonomous cortisol secretion (MACS) will receive osilodrostat to evaluate its effectiveness in normalizing cortisol levels. Evidence suggests that osilodrostat can achieve this, although it might also increase another hormone, ACTH, as a side effect. Overall, the treatment appears promising for managing cortisol-related issues.²⁶⁷⁸⁹