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NCT02697734 or osilodrostat

A Phase III study for people confirmed with persistent or recurrent Cushing’s disease

Patients & Caregivers


Health Care Professionals


Study goal

The goal of this study is to confirm efficacy and safety of osilodrostat for patients with Cushing’s disease who are candidates for medical therapy.

You may be eligible to participate if you

  • Are between 18 and 75 years old
  • Have confirmed Cushing’s disease that is persistent or recurrent as defined by the specific study criteria
  • Discontinued glucocorticoid replacement therapy for at least 7 days or 5 half-lives prior to screening. whichever is longer
  • Have de novo Cushing's disease and are not considered a candidate for surgery (eg, poor surgical candidate due to comorbidities, inoperable tumors, patients who refuse to have surgical treatment, or surgical treatment is not available)
  • Meet other eligibility criteria




Clinical Study Overview

Here is some information that may help you gain a better understanding of this study and its goals. By learning more about this study, you'll be able to make an informed decision with your doctor.

What are the possible drugs that I could receive in this study?

Osilodrostat or placebo.

In which disease is the investigational drug being studied?

The study population will be comprised of approximately 69 adult male and female patients with Cushing’s disease who have persistent or recurrent hypercortisolism after primary pituitary surgery and/or irradiation, and patients with de novo Cushing’s disease who are not surgical candidates for medical reasons, or refuse to undergo surgery, or do not have access to a specialized center with experience in pituitary surgery.

What should I know about the investigational drug in this study?

The investigational drug, osilodrostat, is given as a tablet. Efficacy and safety have not been confirmed and approved by the U.S. Food and Drug Administration (FDA). There is no guarantee that osilodrostat will become commercially available.

It has not been approved by the FDA as a safe and effective treatment for this condition.

How many patients will participate in this study?

Approximately 69 people in several countries around the world will participate in this study.

How long will I participate in this study?

This study is a clinical research study consisting of two periods with a total expected duration of 48 weeks, followed by an optional extension phase for an additional 52 weeks. Dosing with the study drug will continue as long as the study doctor thinks you may be benefiting from the study drug, or until you experience unacceptable side effects, or until discontinuation from the study drug for any other reason, or until the study has concluded.


To learn more about this study, visit