Cushing DiseasePhase 2NCT05971758
Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease
A Phase 2 study testing Fimepinostat in people with Cushing Disease, run by University of California, Los Angeles at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cushing Disease
- What is being tested?
- Fimepinostat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Jan 2025to Jan 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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:
- 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.
- Recurrent or persistent CD defined as pathologically confirmed previously resected pituitary ACTH-secreting tumor, and 24 hour UFC >ULN at least 4 weeks after pituitary surgery.
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- Patients on medical treatment for CD. Washout periods will be completed as below before screening: Inhibitors of steroidogenesis (metyrapone, ketoconazole, osilodristat,
- Levo-ketoconazole): 2 weeks
- SRLs (pasireotide): 2 weeks
- Progesterone receptor antagonist (mifepristone): 2 weeks
- Dopamine agonists (cabergoline): 4 weeks
- CYP3A4 strong inducers or inhibitors: varies between drugs; minimum 5-6 times the half-life of drug
Who cannot join
- 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
- Patients with serum potassium < 3.5 mEq/L unless stably controlled on potassium supplementation
- Patients with poorly-controlled Diabetes mellitus evidenced by HbA1c levels >8
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- Patients with poorly controlled hypertension (i.e. blood pressure ≥ 160/100 mm Hg)
- Patients who have clinically significant cardiovascular impairment, as evidenced by the presence of bradycardia, ventricular tachycardia, history of myocardial infarction within past year, or any other cardiovascular impairment that may pose significant health risk in view of the investigator.
- Patients with liver disease or history of liver disease such as cirrhosis, chronic active hepatitis B and C, or chronic persistent hepatitis, or patients with ALT or AST >1.5 x ULN, serum total bilirubin >ULN, serum albumin <0.67 x LLN at screening
- Patients with renal disease or history of renal disease with creatinine clearance of 30 cm3/min or less and/or creatinine > 1.5 mg/dl at screening
- Patients not biochemically euthyroid. Patients receiving thyroid-replacement therapy must be on a stable dose for at least 3 months.
- Patients who are known to be positive for HIV, or any other condition that significantly compromises subject's immune system.
- History of alcohol abuse or illicit substance use within past year.
- Female patients who are pregnant or lactating or are of childbearing potential unless willing to practice acceptable method of birth control. Women participating in the trial must employ double barrier method through oral contraceptive or diaphragm with partner utilizing a condom. Abstinence is an acceptable form of birth control if routinely practiced. Male participants must utilize a condom with spermicidal cap/jelly and agree to not donate sperm for up to 3 months beyond main study period.
- Patients who have participated in any clinical investigation with an investigational drug within 1 month prior to screening or within 5 half-lives of the investigational treatment whichever is longer.
- Patients with concomitant treatment of strong CYP3A4 inducers or inhibitors.
- Patients who have received pituitary irradiation within the last 5 years prior to the baseline visit
- Patients with known hepatitis B surface antigen (HbsAg) positivity
- Patients with known hepatitis C antibody (anti-HCV) positivity
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Ronald Reagan Medical Center
Los Angeles, California, 90095
About this study
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.
- Conditions
- Cushing Disease
- Being tested
- Fimepinostat
- Medical subject headings
- Pituitary ACTH Hypersecretion
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FimepinostatDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Fimepinostat 60mg | Active comparator | two 30mg capsules once a day, 10 subjects Receives: Drug: Fimepinostat |
| Fimepinostat 30mg | Active comparator | single 30mg capsule daily in 10 subjects Receives: Drug: Fimepinostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline, 4 weeks
Secondary outcomes (6)
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
Measured: Baseline, 4 weeks
body weight change from baseline
Measured: Baseline, Day 28
body mass index change from baseline
Measured: Baseline, Day 28
Systolic and Diastolic Blood Pressure Change from baseline
Measured: Baseline, Day 28
Cushing Disease health-related quality of life questionnaire (CushingQoL) change from baseline
The CushingQoL is scored using a total score ranging from 0 to 100 with lower scores indicating a greater impact on QoL
Measured: Baseline, Day 28
Beck Depression inventory second edition (BDIII) change from baseline
Beck Depression scale is scored as follows 1-10 These ups and downs are considered normal. 11-16 Mild mood disturbances. 17-20 borderline clinical depression. 21-30 Moderate depression. 31-40 Severe depression
Measured: Baseline, Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2023
- Study start16 Jan 2025
- Primary completionJan 2029 (estimated)
- Study completionJan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cushing DiseaseStudies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Anthony P. Heaney, University of California, Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05971758
- Sponsor's protocol ID
- FD-07548-01-A1
- Other IDs
- R01FD007548-01A1
- Status last verified
- Jul 2026
- First submitted
- 6 Jul 2023
- Last updated
- 17 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease
- ClinicalTrials.gov ID
- NCT05971758
- Acronym / sponsor ID
- FD-07548-01-A1
- Conditions
- Cushing Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- Study start
- 16 Jan 2025 (actual)
- Primary completion
- Jan 2029 (estimated)
- Study completion
- Jan 2029 (estimated)
- First posted
- 2 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Fimepinostat 60mg | Active comparator | Drug: Fimepinostattwo 30mg capsules once a day, 10 subjects |
| Fimepinostat 30mg | Active comparator | Drug: Fimepinostatsingle 30mg capsule daily in 10 subjects |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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:
Key exclusion criteria (19 listed)
- 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
- Patients with serum potassium < 3.5 mEq/L unless stably controlled on potassium supplementation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FimepinostatThe 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. | Drug | Fimepinostat 30mg, Fimepinostat 60mg |
Primary outcome
Number of participants with mUFC ≤ 1.0xULN
Time frame: Baseline, 4 weeks
4 week UFC calculated as the mean of three 24h UFC specimens collected on consecutive days between day 24-28
6 secondary outcomes
Number of participants with normalization (values within normal limits) or >50% improvement from baseline in 24h UFC, plasma ACTH, serum and salivary cortisol levels
Time frame: Baseline, 4 weeks
body weight change from baseline
Time frame: Baseline, Day 28
body mass index change from baseline
Time frame: Baseline, Day 28
Systolic and Diastolic Blood Pressure Change from baseline
Time frame: Baseline, Day 28
Cushing Disease health-related quality of life questionnaire (CushingQoL) change from baseline
Time frame: Baseline, Day 28
Beck Depression inventory second edition (BDIII) change from baseline
Time frame: Baseline, Day 28
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fimepinostat in people with Cushing Disease, run by University of California, Los Angeles at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Anthony Heaney, MD (310-825-5874, aheaney@mednet.ucla.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with mUFC ≤ 1.0xULN (time frame: Baseline, 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jul 2026.