New treatment for Cushing Disease using a combination therapy

Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease (IND 168501 - 07/31/2023)

NIH-funded research University of California Los Angeles · NIH-10973945

This study is testing a new medication called Fimepinostat to see if it can help people with Cushing Disease, which is caused by a tumor in the pituitary gland, by looking at how safe and effective it is over four weeks of treatment.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of California Los Angeles NIH-funded
Lab location1 site (Los Angeles, United States)
Project IDNIH-10973945 on NIH RePORTER

What this research studies

This research investigates the use of Fimepinostat, a dual inhibitor targeting specific pathways involved in Cushing Disease, which is caused by a tumor in the pituitary gland. The study will involve a short-term treatment period of four weeks, where patients will receive either a higher or lower dose of the medication to evaluate its safety and effectiveness. Patients will be closely monitored for changes in cortisol levels and overall health during the trial. The goal is to provide a new therapeutic option for individuals suffering from this rare and serious condition.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals diagnosed with de novo, persistent, or recurrent Cushing Disease.

Not a fit: Patients with other types of pituitary disorders or those who do not have Cushing Disease may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to a new effective treatment for patients with Cushing Disease, potentially improving their quality of life and reducing healthcare costs.

How similar studies have performed: While this approach is novel in the context of Cushing Disease, similar combination therapies have shown promise in other tumor types, suggesting potential for success.

Where this research is happening

Los Angeles, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.