University of California, Los Angeles
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.
Inclusion Criteria: * Male and female patients at least 18 years old * Patients with confirmed pituitary origin Cushing syndrome defined as 1, 2\& 3 or 4 \& 5 below: 1. Persistent hypercortisolism defined as a mean of 3 consecutive 24h UFC at baseline assessment ≥ 1.3x ULN 2. Normal or elevated plasma ACTH levels 3. 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. 4. Recurrent or persistent CD defined as pathologically confirmed previously resected pituitary ACTH-…
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.
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