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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a drug called Fimepinostat to determine its safety and effectiveness in treating Cushing's Disease, a condition where the body produces excess cortisol, often due to a pituitary tumor. Researchers will test two different doses of the drug to identify the more effective option. Suitable candidates for this trial include those with Cushing's Disease from a pituitary tumor who continue to experience symptoms or have had symptoms return after treatment. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants, providing them an opportunity to contribute to significant advancements in treatment.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that Fimepinostat is under investigation for safety in treating Cushing's Disease. In earlier studies, patients generally tolerated Fimepinostat well. Common side effects included nausea and tiredness, but these were usually mild.

The current study tests two doses—30mg and 60mg—to determine if different doses have varying safety levels. As a Phase 2 trial, the treatment has shown some safety in earlier studies. The goal is to better understand its safety and effectiveness in a larger group.

For those considering joining this trial, it's important to know that while the treatment is still being tested, it has been deemed safe enough to reach this stage of research. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for Cushing's Disease, which often focus on reducing cortisol production or blocking its effects, Fimepinostat offers a unique approach. It targets specific cellular pathways involved in the disease, potentially addressing the root cause rather than just the symptoms. Researchers are excited about Fimepinostat because it uses a novel mechanism that could provide a more effective and targeted treatment option, potentially leading to better outcomes for patients with fewer side effects. This innovative approach could make a significant difference in managing Cushing's Disease.

What evidence suggests that this trial's treatments could be effective for Cushing's Disease?

Research shows that Fimepinostat could be a promising treatment for Cushing's Disease. The trial will study two different dosages of Fimepinostat: 60mg and 30mg. This drug blocks certain processes in the body that contribute to the disease, specifically targeting areas linked to tumor growth. Early findings suggest that Fimepinostat can help reduce tumor activity and lower cortisol levels, a major concern in Cushing's Disease. Previous studies on similar treatments have shown positive effects in reducing symptoms and improving patients' quality of life. While more research is needed, these early results encourage those considering participation in a clinical trial. ^{1 3 4 5 6}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05971758

Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor ... ... Fimepinostat is an ideal candidate drug in the treatment and intervention of patients with Cushing Disease. The investigators propose a pilot, short-term (4 ...

2. withpower.com withpower.com/trial/phase-2-acth-secreting-pituitary-adenoma-2024-eae6b

Fimepinostat for Cushing's Disease 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 ...

3. ucla.clinicaltrials.researcherprofiles.org ucla.clinicaltrials.researcherprofiles.org/cushings-syndrome

UCLA Cushing's Syndrome Clinical Trials — Los Angeles Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease. open to eligible people ages 18 years and up.

4. trialx.com trialx.com/clinical-trials/listings/condition/651/cushings-disease/

Cushing's Disease Clinical Research Trials Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease. Supported by the pre-clinical data (summarized in Research ...

5. centerwatch.com centerwatch.com/clinical-trials/listings/condition/651/cushings-disease

Cushing's Disease Clinical Research Trials Up to 13 subjects will be treated with 80 mg each day for 4 weeks. The study will also evaluate effects of seliciclib on quality of life and clinical signs and ...

6. ancora.ai ancora.ai/it/details/NCT05971758

Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor ... ... Cushing Disease. The investigators propose a pilot, short-term (4 weeks) phase II single-center study to demonstrate the safety and efficacy of Fimepinostat ...