Pilot Study of Two Different Doses of Fimepinostat for Treating Cushing Disease

What this Trial Is About

Researchers are evaluating Fimepinostat, a combination HDAC and Pi3-kinase inhibitor, as a treatment for patients with Cushing Disease CD, including those with new, persistent, or recurrent disease. This pilot Phase II study is designed to assess the safety and effectiveness of two different doses of Fimepinostat in a short-term 4-week treatment period. The study aims to guide dose selection for future larger trials and is sponsored by the University of California, Los Angeles with funding from the FDA Office of Orphan Products Development. Participants will be randomly assigned to receive either two 30mg capsules once daily or one 30mg capsule once daily of Fimepinostat. The study has a two-arm design with 10 subjects in each dosing group. Treatment lasts for 4 weeks, with the goal to evaluate the drugs impact on biochemical markers and clinical outcomes relevant to Cushing Disease. During the study, participants will undergo assessments including measurement of urinary free cortisol, plasma ACTH, serum and salivary cortisol levels, body weight, body mass index, blood pressure, and quality of life questionnaires related to Cushing Disease and depression. These evaluations occur at baseline and after 4 weeks of treatment to monitor efficacy and safety. The primary outcome is the number of participants achieving normalized urinary free cortisol levels. The total study duration for participants is approximately one month.

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