Fimepinostat for Cushing's Disease

Overseen By Cristian Santana, BS

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of California, Los Angeles

Must not be taking: CYP3A4 inducers/inhibitors

Disqualifiers: Visual changes, Diabetes, Hypertension, Cardiovascular, Liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 28

Duration: 4 weeks

20 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

Yes, if you are taking certain medications for Cushing's Disease, you will need to stop them before joining the trial. There is a 'washout period' (time without taking certain medications) of 2 to 4 weeks for specific drugs like metyrapone, ketoconazole, and cabergoline, among others.

Eligibility Criteria

This trial is for adults with Cushing Disease who have high cortisol levels, a history of pituitary ACTH-secreting tumors, and visible adenoma on MRI or specific test results. Patients already on treatment must undergo washout periods before joining.

Inclusion Criteria

You have an average of 3 24-hour urinary free cortisol concentrations higher than 1.3 times the upper limit of normal at initial evaluation.

Your plasma ACTH levels are either normal or elevated.

You have a pathologically confirmed ACTH-secreting tumor that was recently resected, and your 24 hour UFC is greater than the upper limit of normal at least four weeks after pituitary surgery.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Fimepinostat for 4 weeks to assess safety and efficacy in treating Cushing Disease

4 weeks

Baseline, Day 28

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Fimepinostat

Trial Overview The study tests Fimepinostat's safety and effectiveness in treating Cushing Disease at UCLA. It's a phase II trial lasting 4 weeks, comparing two doses to find the best one for future research.

Participant Groups

Group I: Fimepinostat 60mg Active Control 1 Intervention

two 30mg capsules once a day, 10 subjects

Group II: Fimepinostat 30mg Active Control 1 Intervention

single 30mg capsule daily in 10 subjects

Who Is Running the Clinical Trial?

University of California, Los Angeles

Lead Sponsor

Trials

1,594

Recruited

10,430,000+