RecruitingPhase 2NCT05971758

Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease

Sponsor

University of California, Los Angeles

Enrollment

20 participants

Start Date

Jan 16, 2025

Study Type

INTERVENTIONAL

Conditions

Cushing Disease

Summary

Supported by the pre-clinical data (summarized in Research Strategy), the investigators propose that Fimepinostat is an ideal candidate drug in the treatment and intervention of patients with Cushing Disease. The investigators propose a pilot, short-term (4 weeks) phase II single-center study to demonstrate the safety and efficacy of Fimepinostat in the treatment of patients with de novo, persistent, and/or recurrent CD recruited at the University of California, Los Angeles. The trial will have a 2-arm design and will simultaneously examine two different doses of Fimepinostat. The study will allow the investigators to determine the efficacy and safety of these doses in the treatment of CD and guide dose selection for subsequent, larger studies. Funding Source - FDA OOPD.

Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a drug called fimepinostat — which inhibits two enzymes involved in cancer cell survival — in people with Cushing's disease. Cushing's disease is caused by a benign tumor in the pituitary gland (at the base of the brain) that produces too much of a hormone called ACTH, which in turn causes the adrenal glands to overproduce cortisol (the stress hormone). Too much cortisol causes weight gain, high blood pressure, diabetes, and many other serious problems. This trial tests whether fimepinostat can reduce cortisol levels. **You may be eligible if...** - You are 18 years or older - You have confirmed Cushing's disease caused by a pituitary ACTH-secreting tumor - Your cortisol levels are confirmed to be significantly elevated through a 24-hour urine test - Your ACTH levels are normal or elevated, and imaging or a special vein sampling test confirms pituitary origin - You have had persistent or recurrent disease after pituitary surgery, or are on medical treatment for Cushing's disease **You may NOT be eligible if...** - You have vision problems caused by your pituitary tumor pressing on the optic nerve - Your Cushing's syndrome is caused by something other than a pituitary tumor (e.g., adrenal tumor or ectopic ACTH) - You have recently had major surgery - You are pregnant or breastfeeding Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGFimepinostat

The study will allow us to determine the efficacy and safety of these doses in the treatment of Cushing Disease (CD) and guide dose selection for subsequent, larger studies.