Phase 3 Trial Shows Positive Results for Investigational Therapy for Cushing Syndrome

A Phase 3 trial for Cushing syndrome led by Maria Fleseriu at Oregon Health and Science University has just been completed. It examined a new investigational treatment called levoketoconazole.

About the Trial

This Phase 3 trial was non-randomized and open-label. It included 94 patients diagnosed with Cushing Syndrome. For these participants, cortisol levels were 4.9 times the normal level. Patients were given the investigational medication twice each day by oral route, starting at 150mg, and then increasing during a titration phrase in effort to normalize cortisol levels.


The primary outcome of the investigation was the proportion of participants who were able to maintain normal cortisol levels at the end of a 6 month maintenance phase.

  • 81% of patients experienced normalized cortisol levels
  • Normalized cortisol levels were sustained for 6 months for 31% of patients
  • Improvements were documented in cardiovascular risk factors including reductions in-
    • LDL cholesterol
    • Glycated hemoglobin
    • Body weight
  • No improvements were documented for blood pressure
  • A slight decrease was noted in good cholesterol

Researchers emphasize the cardiovascular findings of this investigation as cardiovascular disease is responsible for much of the mortality associated with Cushing syndrome. By examining changes in cardiovascular risk markers, the team was able to confidently predict this therapies ability to prevent serious cardiovascular adverse events.

It is important to note that 12 patients discontinued the study due to serious adverse events. However, the most common AEs were nausea and headache.

Limitations and Comparisons

Typically, Cushing syndrome is treated with surgery, however this option is far from perfect. When surgery is delayed or unsuccessful, ketoconazole is typically prescribed. This medication has not been approved in the United States however it is approved for the disease in the U.K.

Levoketoconazole is a more potent drug than ketoconazole and researchers believe it could be used at a lower dose.

Unfortunately, these two medications have not been compared in a head to head trial because recruitment for participants is difficult with such a small population of patients. This is certainly a limitation of this study. However, nonetheless, this investigation does show the potential of Levoketoconazole as a potential therapeutic option for patients.

You can read more about this Phase 3 investigation and potential new medication for Cushing syndrome here.

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