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.
Male and female patients at least 18 years old
Persistent hypercortisolism defined as a mean of 3 consecutive 24h UFC at baseline assessment ≥ 1.3x ULN
Normal or elevated plasma ACTH levels
Pituitary adenoma > 4mm visible on MRI or inferior petrosal sinus sampling (IPSS) central to peripheral ACTH gradient >2 at baseline and/or >2 after DDAVP stimulation.
Patients with compromised visual fields, or evidence of visual changes within past 6 months
Patients with sellar tumor abutting or compressing the optic chiasm on MRI and normal visual fields
Patients with Cushing's syndrome not due to an ACTH-secreting pituitary tumor
Patients who have undergone major surgery including pituitary surgery within 1 month of screening or who have any major surgical procedures planned across the study period
Single group
Fimepinostat
DrugThe 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.
Number of participants with mUFC ≤ 1.0xULN
4 week UFC calculated as the mean of three 24h UFC specimens collected on consecutive days between day 24-28
Number of participants with normalization (values within normal limits) or >50% improvement from baseline in 24h UFC, plasma ACTH, serum and salivary cortisol levels
4 week UFC calculated as the mean of three 24h UFC specimens collected on consecutive days between day 24-28
body weight change from baseline
body mass index change from baseline
Systolic and Diastolic Blood Pressure Change from baseline
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