Actively Recruiting

Phase 2
Age: 18Years +
All Genders
Healthy Volunteers
NCT05971758

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

Led by University of California, Los Angeles · Updated on 2025-07-04

20

Participants Needed

1

Research Sites

206 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Male and female patients at least 18 years old
  • Patients with confirmed pituitary origin Cushing syndrome defined by all of the following:
    1. Persistent hypercortisolism with mean of 3 consecutive 24h urine free cortisol (UFC) at baseline  1.3 times upper limit of normal (ULN)
    2. Normal or elevated plasma ACTH levels
    3. Pituitary adenoma larger than 4mm visible on MRI or inferior petrosal sinus sampling (IPSS) showing central to peripheral ACTH gradient >2 at baseline and/or >2 after DDAVP stimulation
    4. Recurrent or persistent CD with pathologically confirmed previously resected pituitary ACTH-secreting tumor and 24h UFC > ULN at least 4 weeks after pituitary surgery
    5. Patients on medical treatment for CD who have completed required washout periods before screening: 2 weeks for steroidogenesis inhibitors (metyrapone, ketoconazole, osilodristat, levo-ketoconazole), 2 weeks for SRLs (pasireotide), 2 weeks for progesterone receptor antagonist (mifepristone), 4 weeks for dopamine agonists (cabergoline), and minimum 5-6 times half-life for CYP3A4 strong inducers or inhibitors
Not Eligible

You will not qualify if you...

  • Patients with compromised visual fields or recent visual changes within past 6 months
  • Patients with sellar tumor touching or compressing the optic chiasm on MRI but normal visual fields
  • Patients with Cushing's syndrome not caused by an ACTH-secreting pituitary tumor
  • Patients who had major surgery including pituitary surgery within 1 month of screening or planned major surgery during study
  • Patients with serum potassium less than 3.5 mEq/L unless stably controlled with supplements
  • Patients with poorly controlled diabetes mellitus (HbA1c > 8)
  • Patients with poorly controlled high blood pressure (>= 160/100 mm Hg)
  • Patients with significant cardiovascular problems such as bradycardia, ventricular tachycardia, recent heart attack, or other health risks
  • Patients with liver disease or abnormal liver tests exceeding specified limits
  • Patients with kidney disease or creatinine clearance 30 cm3/min or less, or creatinine > 1.5 mg/dl
  • Patients not biochemically euthyroid or on unstable thyroid replacement therapy
  • Patients positive for HIV or with significant immune system compromise
  • History of alcohol abuse or illicit substance use within past year
  • Female patients who are pregnant, breastfeeding, or of childbearing potential not using required birth control methods; male patients must use condom with spermicide and avoid sperm donation for 3 months post-study
  • Patients who participated in other investigational drug studies within 1 month or 5 half-lives of treatment
  • Patients on strong CYP3A4 inducers or inhibitors
  • Patients who had pituitary irradiation within last 5 years
  • Patients positive for hepatitis B surface antigen or hepatitis C antibody

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Ronald Reagan Medical Center

Los Angeles, California, United States, 90095

Actively Recruiting

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Research Team

A

Anthony Heaney, MD

CONTACT

C

Cristian Santana, BS

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

2

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