Actively Recruiting
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
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating Fimepinostat, a combination HDAC and Pi3-kinase inhibitor, as a potential treatment for patients with Cushing Disease (CD). This pilot, short-term phase II study aims to assess the safety and effectiveness of two different doses of Fimepinostat in adults with new, persistent, or recurrent CD. The study is supported by pre-clinical data and intends to guide dose selection for future larger trials. The trial has a randomized design with two groups: one receiving two 30mg capsules daily and the other receiving a single 30mg capsule daily. The treatment period lasts for 4 weeks. Participants will receive the study drug orally and researchers will monitor the effects of both doses to determine safety and efficacy. During the study, participants will undergo evaluations including measurement of urinary free cortisol levels, plasma ACTH, serum and salivary cortisol, body weight, body mass index, and blood pressure. They will also complete questionnaires assessing health-related quality of life and depression symptoms. Safety monitoring and follow-up assessments will be conducted throughout the 4-week treatment period.
CONDITIONS
Brief Title
Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female patients at least 18 years old
- Confirmed pituitary origin Cushing syndrome with persistent hypercortisolism (mean of 3 consecutive 24h UFC 1.3x ULN)
- Normal or elevated plasma ACTH levels
- Pituitary adenoma > 4mm visible on MRI or IPSS central to peripheral ACTH gradient >2 at baseline or after DDAVP stimulation
- Recurrent or persistent Cushing Disease with pathologically confirmed pituitary ACTH-secreting tumor and 24h UFC > ULN at least 4 weeks after pituitary surgery
- Patients on medical treatment for Cushing Disease with required washout periods completed before screening
You will not qualify if you...
- Compromised visual fields or visual changes within past 6 months
- Sellar tumor abutting or compressing optic chiasm on MRI with normal visual fields
- Cushing's syndrome not due to ACTH-secreting pituitary tumor
- Major surgery including pituitary surgery within 1 month before screening or planned during study
- Serum potassium < 3.5 mEq/L unless stably controlled on supplementation
- Poorly controlled Diabetes mellitus (HbA1c > 8)
- Poorly controlled hypertension (blood pressure 160/100 mm Hg)
- Clinically significant cardiovascular impairment
- Liver disease or abnormal liver function tests above defined limits
- Renal disease with creatinine clearance 30 cm3/min or creatinine > 1.5 mg/dl
- Not biochemically euthyroid or unstable thyroid therapy
- Known HIV or significant immune compromise
- History of alcohol abuse or illicit substance use within past year
- Pregnant or lactating women or women of childbearing potential not using effective contraception
- Participation in other investigational drug trials within 1 month or 5 half-lives of treatment
- Concomitant treatment with strong CYP3A4 inducers or inhibitors
- Pituitary irradiation within last 5 years prior to baseline
- Known hepatitis B surface antigen or hepatitis C antibody positivity
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 4 weeks
Participants receive Fimepinostat drug therapy to evaluate efficacy and safety for Cushing Disease.
Daily dosing with either two 30mg capsules or one 30mg capsule
Trial Site Locations
Total: 1 location
1
Ronald Reagan Medical Center
Los Angeles, California, United States, 90095
Actively Recruiting
Research Team
A
Anthony Heaney, MD
C
Cristian Santana, BS
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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