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Levoketoconazole

Phase 3

Cushing Syndrome | Small molecule | Endocrine |Xeris Biopharma Holdings, Inc.|Last Updated: Jun 19, 2025

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment51

FDA Designations

No designations recorded

Clinical trial landscape

Levoketoconazole · 5 trials · 5 indications

Phase 3 3Phase 1 2
NCT03621280Open-label Treatment in Cushing's SyndromeCushing Syndrome
COMPLETED51 Analytics
NCT03277690A Study to Assess the Safety and Efficacy of Levoketoconazole in the Treatment of Endogenous Cushing's Syndrome.Endogenous Cushing's Syndrome
COMPLETED84 Analytics
NCT01838551Treatment for Endogenous Cushing's SyndromeEndogenous Cushing's Syndrome
COMPLETED94 Analytics
PHASE3COMPLETED
Open-label Treatment in Cushing's Syndrome
Cushing SyndromeUnlock trial analytics
PHASE3COMPLETED
A Study to Assess the Safety and Efficacy of Levoketoconazole in the Treatment of Endogenous Cushing's Syndrome.
Endogenous Cushing's SyndromeUnlock trial analytics
PHASE3COMPLETED
Treatment for Endogenous Cushing's Syndrome
Endogenous Cushing's SyndromeUnlock trial analytics

Study Endpoints

Primary Endpoints

Proportions of Participants With Mean Urinary Free Cortisol (mUFC) Categorization Based on Upper Limit of Normal (ULN)
From parent study Baseline to final study visit or up to a maximum of 3 years, whichever comes first.

Proportions of participants with mUFC: 1) Less or equal to the ULN of the reference range, 2) Above the ULN to 1.5x the ULN, and 3) Above 1.5x the ULN.

Number of Subjects With Loss of Therapeutic Response to Levoketoconazole Upon Withdrawing to Placebo Compared With the Proportion of Subjects With Loss of Therapeutic Response Upon Continuing Treatment With Levoketoconazole.
max. 9.5 weeks

Urinary free cortisol (UFC) level measurement: Loss of therapeutic response is inferred based on mUFC from three 24-hour UFC measurements obtained at any visit from second through final Randomized Withdrawal Phase visits (RW1 through RW5 inclusive) when: (1) mUFC is above 1.5X the ULN of the central laboratory's reference range, OR (2) mUFC is more than 40% above the baseline (RW0) value, if the RW0 value is above the ULN (i.e. \>1.0X ULN)1, OR (3) an early rescue criterion is met.

Normalization in Urinary Free Cortisol in Patients With Endogenous Cushing's Syndrome.
6 months of maintenance phase therapy without a prior dose increase during that phase

The response to COR-003 is defined as mean UFC concentration ≤ULN following 6 months of maintenance phase therapy without a prior dose increase during that phase. The proportion of responders at the End of Maintenance Phase visit, following 6 months of treatment in the Maintenance Phase, for all dose groups combined was estimated using a generalized linear model with repeated measurements based on a binomial distribution with a logit link function and with region (US vs. non-US), concurrent CS medical conditions (diabetes \[Yes/No\], hypertension \[Yes/No\]), age (rounded median split based on the ITT population), sex, disease duration (years), prior CS medication (Yes/No), prior radiation therapy (Yes/No) as Baseline covariates and visit as an independent factor. The least squares mean (LSMEAN) estimate of the UFC response after 6 months of treatment in the Maintenance Phase alongside its 95% Wald CI is presented.

Ratio of geometric least squares means for AUClast of plasma levoketoconazole (2S,4R-ketoconazole)
24 hours

Point estimates and 90 percent (%) confidence intervals (CIs) for the dose-normalized ratios of geometric least squares means between Test (levoketoconazole) and Reference (ketoconazole)

Ratio of geometric least squares means for AUCinf of plasma levoketoconazole (2S,4R-ketoconazole)
24 hours

Point estimates and 90 percent (%) confidence intervals (CIs) for the dose-normalized ratios of geometric least squares means between Test (levoketoconazole) and Reference (ketoconazole)

Ratio of geometric least squares means for Cmax of plasma levoketoconazole (2S,4R-ketoconazole)
24 hours

Point estimates and 90 percent (%) confidence intervals (CIs) for the dose-normalized ratios of geometric least squares means between Test (levoketoconazole) and Reference (ketoconazole)

Maximum observed plasma concentration (Cmax) of levoketoconazole
24 hours

Maximum observed plasma concentration (Cmax) of levoketoconazole for fed vs. fasted condition

Time to maximum concentration (Tmax) of levoketoconazole
24 hours

Time to maximum concentration (Tmax) of levoketoconazole for fed vs. fasted condition

Apparent Terminal Elimination Phase Rate Constant (λz) of levoketoconazole
24 hours

Apparent Terminal Elimination Phase Rate Constant (λz) of levoketoconazole for fed vs. fasted condition

Terminal phase half-life (t 1/2) of levoketoconazole
24 hours

Terminal phase half-life (t 1/2) of levoketoconazole for fed vs. fasted condition

Area under the plasma concentration-time curve (AUC) of levoketoconazole
24 hours

Area under the plasma concentration-time curve (AUC) from time 0 to time of last measurable plasma concentration (AUClast) and from time 0 extrapolated to infinity (AUCinf) of levoketoconazole for fed vs. fasted condition

Secondary Endpoints

Terminal phase rate constant (λz)
24 hours
Time to Maximum Plasma concentration (Tmax)
24 hours
Lag-time (Tlag)
24 hours
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
LevoketoconazoleEXPERIMENTALLevoketoconazole taken twice daily up to 1200 mg daily
PlaceboPLACEBO_COMPARATORDouble blind withdrawal phase: Placebo; Double blind restoration phase: Placebo plus Levoketoconazole
Levoketoconazole DL0EXPERIMENTALLevoketoconazole Tablets Dose Level 0 Once Daily
Levoketoconazole DL1EXPERIMENTALLevoketoconazole Tablets Dose Level 1 Twice Daily
Levoketoconazole DL2EXPERIMENTALLevoketoconazole Tablets Dose Level 1 Twice Daily
Levoketoconazole DL3EXPERIMENTALLevoketoconazole Tablets Dose Level 3 Twice Daily
Levoketoconazole DL4EXPERIMENTALLevoketoconazole Tablets Dose Level 4 Twice Daily
Levoketoconazole DL5EXPERIMENTALLevoketoconazole Tablets Dose Level 5 Twice Daily
Levoketoconazole DL6EXPERIMENTALLevoketoconazole Tablets Dose Level 6 Twice Daily
Levoketoconazole DL7EXPERIMENTALLevoketoconazole Tablets Dose Level 7 Twice Daily
KetoconazoleACTIVE_COMPARATORKetoconazole 200 mg
Fasting StateOTHERA single 600 mg dose of levoketoconazole administered in a fasting state.
Fed StateOTHERA single 600 mg dose of levoketoconazole administered in a fed state.

Interventions

NameTypeDescription
LevoketoconazoleDRUGLevoketoconazole up to 1200 mg daily
PlaceboDRUGDuring the double-blind Withdrawal Phase, patients will receive placebo tablets. During the double-blind Restoration Phase, patients will receive placebo plus levoketoconazole.
KetoconazoleDRUGKetoconazole tablet
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites30

Key Inclusion Criteria: 1. Completed the Extended Evaluation Phase of Study COR-2012-01 (i.e. M12) 2. Completed the Restoration Phase of Study COR-2017-01 (i.e. RES2) NOTE: Participants meeting criteria 1 or 2 above who have had a break in therapy may be eligible only after discussion with the ...

Countries:United StatesBulgariaFranceGreeceHungaryIsraelItalyNetherlandsPolandRomaniaSpainDenmarkBelgiumCanadaCzechiaGermanySerbiaTurkey (Türkiye)
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Frequently asked questions about Levoketoconazole

What is Levoketoconazole used for?

Levoketoconazole is an investigational small molecule being developed for the treatment of endogenous Cushing's syndrome, a condition caused by excess cortisol production. It is also being studied in healthy subjects for bioavailability purposes. The drug is currently in Phase 3 clinical development and has not been approved by regulatory authorities.

Who makes Levoketoconazole?

Levoketoconazole is being developed by Xeris Biopharma Holdings, Inc., a company traded on the stock exchange under the ticker symbol XERS. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with endogenous Cushing's syndrome.

What phase is Levoketoconazole in?

Levoketoconazole is in Phase 3 clinical development for endogenous Cushing's syndrome. It remains an investigational drug and has not received FDA approval. Several Phase 3 trials have been completed, but the drug is still undergoing clinical evaluation to determine its safety and effectiveness.

What clinical trials is Levoketoconazole in?

Levoketoconazole has been studied in several completed clinical trials, including NCT01838551 and NCT03277690, both Phase 3 studies in endogenous Cushing's syndrome. An additional Phase 3 open-label trial, NCT03621280, and a Phase 1 bioavailability study, NCT04212000, have also been completed. These trials enrolled a total of 178 participants.

How does Levoketoconazole work?

Levoketoconazole is a small molecule that targets cortisol production. It works by inhibiting enzymes involved in steroidogenesis, thereby reducing cortisol levels in the body. This mechanism is intended to address the underlying hormonal imbalance in endogenous Cushing's syndrome.

Is Levoketoconazole the same as ketoconazole?

Levoketoconazole is a distinct drug from ketoconazole, though they are related compounds. A Phase 1 study (NCT04212000) compared the bioavailability of levoketoconazole and ketoconazole tablets in healthy subjects. Levoketoconazole is being developed specifically for endogenous Cushing's syndrome.