CLINICAL TRIALS PROFILE FOR KETOCONAZOLE

505(b)(2) Clinical Trials for ketoconazole

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT01110330 ↗	An Efficacy Study of a New Formulation of Ketoconazole 2% Cream in Patients With Tinea Pedis, Commonly Known as Athlete's Foot	Terminated	Johnson & Johnson Pharmaceutical Research & Development, L.L.C.	Phase 3	2007-07-01	The purpose of this study is to determine if a new formulation of ketoconazole 2% cream is as effective as a current formulation of ketoconazole 2% cream (Nizoral) compared with placebo in treating patients with Tinea pedis, a skin infection commonly known as "athlete's foot" that is caused by a kind of mold called a fungus.
OTC	NCT03513393 ↗	Influence of Cola on the Absorption of the HCV Agent Velpatasvir in Combination With PPI Omeprazole.	Completed	Radboud University	Phase 1	2018-08-01	Epclusa® is a pan-genotypic, once-daily tablet for the treatment of chronic hepatitis C virus (HCV) infection containing the NS5B- polymerase inhibitor sofosbuvir (SOF, nucleotide analogue) 400 mg and the NS5A inhibitor velpatasvir (VEL) 100 mg. Velpatasvir has pH dependent absorption. At higher pH the solubility of velpatasvir decreases. It has been shown that in subjects treated with proton pump inhibitors (PPIs) such as omeprazole, the absorption of velpatasvir is reduced by 26-56%, depending on the dose of omeprazole, concomitant food intake, and timing/sequence of velpatasvir vs. omeprazole intake. As a result, concomitant intake of PPIs with velpatasvir is not recommended. For a number of reasons, the prohibition of PPI use with velpatasvir is a clinically relevant problem. First, PPI use is highly frequent in the HCV-infected subject population with prevalences reported up to 40%. Second, PPIs are available as over-the-counter medications and thus can be used by subjects without informing their physician. Third, although HCV therapy is generally well tolerated, gastro-intestinal symptoms such as abdominal pain and nausea are frequently reported, which my lead to PPI use. One solution of this problem could be the use of other acid-reducing agents such as H2-receptor antagonists or antacids. In general, they have a less pronounced effect on intragastric pH, and are considered less effective than PPIs by many patients and physicians. A second solution would be the choice of another HCV agent or combination that is not dependent on low gastric pH for its absorption such as daclatasvir. Daclatasvir, however, is not a pan-genotypic HCV agent and may be less effective against GT 2 and 3 infections than velpatasvir. Second, not all subjects have access to daclatasvir, depending on health insurance company or region where they live. A third solution, and the focus of this COPA study, is to add a glass of the acidic beverage cola at the time of velpatasvir administration in subjects concurrently treated with PPIs. This intervention has been shown to be effective for a number of drugs from other therapeutic classes who all have in common a reduced solubility (and thus reduced absorption) at higher intragastric pH, namely erlotinib, itraconazole, ketoconazole. The advantages of this approach are: (1) only a temporary decrease in gastric pH at the time of cola intake; the rest of the day the PPI will have its therapeutic effect (2) cola is available worldwide (3) the administration of cola can be done irrespective to the timing of PPI use.

All Clinical Trials for ketoconazole

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000579 ↗	Acute Respiratory Distress Syndrome Clinical Network (ARDSNet)	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	1994-09-01	The purposes of this study are to assess rapidly innovative treatment methods in patients with adult respiratory distress syndrome (ARDS) as well as those at risk of developing ARDS and to create a network of interactive Critical Care Treatment Groups (CCTGs) to establish and maintain the required infrastructure to perform multiple therapeutic trials that may involve investigational drugs, approved agents not currently used for treatment of ARDS, or treatments currently used but whose efficacy has not been well documented.
NCT00000975 ↗	A Study of Itraconazole in the Treatment and Prevention of Histoplasmosis, a Fungal Infection, in Patients With AIDS	Completed	Janssen Pharmaceuticals	Phase 2	1969-12-31	To evaluate the feasibility of itraconazole as (1) primary therapy in histoplasmosis and (2) maintenance therapy after completion of primary therapy. To evaluate the effect of therapy of CNS histoplasmosis. To determine if resistance to drug occurs in patients who fail therapy. Histoplasmosis is a serious opportunistic infection in patients with AIDS. Although the clinical response to amphotericin B treatment in the AIDS patients is generally good, administration difficulties and toxicity detract from its usefulness. Oral treatment with ketoconazole overcomes these limitations of amphotericin B, but does not appear to be effective for primary treatment in patients with AIDS. Itraconazole is a triazole compound in which preclinical studies have demonstrated activity against Histoplasmosis capsulatum. Preclinical studies have also shown that itraconazole appears effective in the treatment of histoplasmosis. The frequency of adverse reactions to itraconazole has been low in several studies. Central nervous system (CNS) involvement occurs in up to 20 percent of patients with histoplasmosis, and appears to have a poor response to amphotericin B treatment. Itraconazole has been used successfully in a small number of patients with cryptococcal meningitis, supporting a study of its use in CNS histoplasmosis.
NCT00000975 ↗	A Study of Itraconazole in the Treatment and Prevention of Histoplasmosis, a Fungal Infection, in Patients With AIDS	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	To evaluate the feasibility of itraconazole as (1) primary therapy in histoplasmosis and (2) maintenance therapy after completion of primary therapy. To evaluate the effect of therapy of CNS histoplasmosis. To determine if resistance to drug occurs in patients who fail therapy. Histoplasmosis is a serious opportunistic infection in patients with AIDS. Although the clinical response to amphotericin B treatment in the AIDS patients is generally good, administration difficulties and toxicity detract from its usefulness. Oral treatment with ketoconazole overcomes these limitations of amphotericin B, but does not appear to be effective for primary treatment in patients with AIDS. Itraconazole is a triazole compound in which preclinical studies have demonstrated activity against Histoplasmosis capsulatum. Preclinical studies have also shown that itraconazole appears effective in the treatment of histoplasmosis. The frequency of adverse reactions to itraconazole has been low in several studies. Central nervous system (CNS) involvement occurs in up to 20 percent of patients with histoplasmosis, and appears to have a poor response to amphotericin B treatment. Itraconazole has been used successfully in a small number of patients with cryptococcal meningitis, supporting a study of its use in CNS histoplasmosis.
NCT00000992 ↗	A Study of Itraconazole in Preventing the Return of Histoplasmosis, a Fungal Infection, in Patients With AIDS	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To test the effectiveness of itraconazole in preventing the recurrence of disseminated histoplasmosis in AIDS patients. Histoplasmosis is a serious opportunistic infection in patients with AIDS. Amphotericin B has been used to treat the infection. Although the response to this treatment is generally good, up to 90 percent of AIDS patients who have taken amphotericin B to treat their histoplasmosis infection will have a relapse (that is, they will get the disease again) within 12 months following treatment. Ketoconazole has been used to prevent relapse, but available information suggests that up to 50 percent of AIDS patients relapse even with ketoconazole treatment. A more effective therapy to prevent recurrence is needed. Itraconazole has been used successfully to treat disseminated histoplasmosis in non-AIDS patients and it is hoped that it may be more effective in preventing histoplasmosis relapse.
NCT00002304 ↗	A Comparison of Fluconazole and Ketoconazole in the Treatment of Fungal Infections of the Throat in Patients With Weakened Immune Systems	Completed	Pfizer	N/A	1969-12-31	To compare the safety, tolerance, and effectiveness of fluconazole and ketoconazole in the treatment of candidal esophagitis in immunocompromised patients.
NCT00002760 ↗	Antiandrogen Withdrawal in Treating Patients With Hormone-Refractory Prostate Cancer	Completed	National Cancer Institute (NCI)	Phase 3	1996-08-01	RATIONALE: Antiandrogen withdrawal may be an effective treatment for prostate cancer. PURPOSE: Randomized phase III trial to study the effectiveness of ketoconazole and hydrocortisone for antiandrogen withdrawal in treating men with prostate cancer that is refractory to hormone therapy.

Clinical Trial Conditions for ketoconazole

Condition Name

Condition Name for ketoconazole
Intervention	Trials
Healthy	34
Prostate Cancer	24
Healthy Volunteers	8
Cancer	7

Condition MeSH

Condition MeSH for ketoconazole
Intervention	Trials
Prostatic Neoplasms	35
Tinea	8
Infections	6
Tinea Pedis	6

Clinical Trial Locations for ketoconazole

Trials by Country

Trials by Country for ketoconazole
Location	Trials
United States	399
China	16
Australia	15
United Kingdom	13
Netherlands	13

Trials by US State

Trials by US State for ketoconazole
Location	Trials
Texas	33
California	28
New York	25
Illinois	17
Florida	17

Clinical Trial Progress for ketoconazole

Clinical Trial Phase

Clinical Trial Phase for ketoconazole
Clinical Trial Phase	Trials
PHASE4	2
PHASE1	1
Phase 4	22

Clinical Trial Status

Clinical Trial Status for ketoconazole
Clinical Trial Phase	Trials
Completed	171
Terminated	23
Unknown status	18

Clinical Trial Sponsors for ketoconazole

Sponsor Name

Sponsor Name for ketoconazole
Sponsor	Trials
National Cancer Institute (NCI)	21
GlaxoSmithKline	20
AstraZeneca	8

Sponsor Type

Sponsor Type for ketoconazole
Sponsor	Trials
Other	210
Industry	154
NIH	32

Ketoconazole: Clinical Trials Update, Market Analysis, and Projection

Last updated: April 27, 2026

What is ketoconazole’s current clinical and regulatory footprint?

Ketoconazole is an older antifungal drug with multiple approved formulations and established labels. Across major markets, its development pathway is dominated by (1) reformulation history (topical and oral), (2) safety-driven restrictions for systemic use, and (3) ongoing interest in niche indications where benefit-risk is favorable.

Core clinical posture by formulation

Oral ketoconazole (systemic)

• Clinical activity is constrained by safety concerns that drove label restrictions in many jurisdictions, especially around hepatotoxicity and drug interactions.
• Most “new” clinical work in systemic ketoconazole is replacement therapy, optimization, or comparative positioning rather than broad phase-3 expansion.

Topical ketoconazole (dermatologic)

• Clinical development is more consistent because the safety profile supports routine dermatology use.
• Trials in topical ketoconazole typically focus on formulation equivalence, patient-relevant endpoints in fungal dermatoses, or comparative performance against other antifungals.

Ketoconazole in combination or re-positioning

• Beyond classic antifungal use, ketoconazole has long-standing off-label use in oncology-adjacent endocrine contexts (as an inhibitor of steroidogenesis), but this does not translate into a uniform, global “ketoconazole new drug” development pipeline.
• Where ketoconazole appears in newer trial ecosystems, it is often as comparator or anchor in proof-of-mechanism or regimen studies.

Clinical trial intensity (what typically drives updates)

In practice, ketoconazole trial updates cluster around:

• Formulation and bioequivalence: generic and reformulated products entering established markets.
• Safety monitoring studies: especially when systemic exposure is relevant.
• Indication confirmation: topical dermatology and superficial mycoses where standardized endpoints exist.

What does the market look like today for ketoconazole?

Ketoconazole is a mature antifungal with established demand in both prescription and OTC-adjacent channels depending on jurisdiction. The market is shaped by:

• Aging of originator exclusivity (genericization is the dominant economic force)
• Switching pressure from newer antifungals (e.g., azoles with different dosing convenience or safety perception)
• Label limitations for oral systemic use (reducing addressable volume versus earlier decades)
• Sustained dermatology use for topical products (retains a durable base)

Market segmentation that matters for projections

Channel

• Prescription: dominant for systemic and many topical physician-dispensed dermatology products.
• Retail/OTC: varies by country and product licensing; where available, increases volume stability.

Geography

• Mature adoption in North America and Western Europe.
• Higher relative use in parts of Asia and Latin America where antifungal density and dermatology prevalence support steady category sales.

Formulation

• Topical tends to preserve long-term demand due to favorable safety and routine clinical use.
• Oral systemic has constrained growth and often behaves like a mature, safety-limited niche market.

How should ketoconazole market projections be modeled?

Ketoconazole’s projection should use a “base retention” framework: growth is modest, driven by population and dermatology utilization, while systemic volume is capped by regulatory and safety limitations. The economic profile is dominated by generics, so pricing follows competitive erosion rather than premium value creation.

Projection logic (practical model structure)

A robust projection separates three components:

1) Topical antifungal base

• Expected to remain stable to low-single-digit growth over forecast horizons, driven by dermatology incidence and replacement of older patients with chronic/semi-chronic fungal conditions.
• Price declines are gradual and mostly tied to competitive entries and switching among generics.

2) Oral systemic antifungal niche (where still approved)

• Expected to grow slowly or stay flat in revenue terms because safety restrictions reduce physician adoption.
• Generic penetration lowers price, so unit volume gains may not translate into revenue lift.

3) Indirect demand from safety labeling cycles

• Any tightening of systemic warnings or contraindication language typically reduces systemic share further.
• Any label harmonization or improved risk-management may stabilize use but rarely restores old growth trajectories.

Base-case projection range (directional)

Given ketoconazole maturity and the generic-heavy economics, the base-case projection is:

• Revenue: low to mid single-digit CAGR globally across a medium horizon, with regional variability.
• Units: slightly higher than revenue CAGR due to mix shift (from branded to generics) and channel changes.

What is the clinical trial outlook for ketoconazole over the next cycle?

Ketoconazole’s near-term clinical update stream is most likely to be:

• Equivalence and supportive studies that keep products on-shelf and compliant across markets.
• Dermatology endpoint refinement and comparative performance where regulators and payers require demonstration against standard-of-care.

What you should watch in “new trial” screens

The highest-signal updates are:

• Trials that change dose regimen or exposure for topical products, including vehicle- or penetration-modified formulations.
• Studies that document tolerability in real-world dermatology cohorts.
• Regulatory-linked safety monitoring reports for systemic formulations where those are still actively marketed.

Where are the commercial risks and upside points?

Risks

• Systemic safety restrictions continue to limit addressable demand for oral ketoconazole.
• Competitive displacement: newer antifungals with improved dosing and tolerability can take share.
• Margin compression as new generics enter and price competition intensifies.

Upside points

• Topical durability: chronic fungal and seborrheic conditions support consistent use cycles.
• Formulation improvements: reformulations that improve adherence or reduce irritation can shift mix toward higher utilization even in generic categories.
• Geographic lag: markets that adopt newer antifungals slower can retain ketoconazole demand longer.

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Key Takeaways

• Ketoconazole is a mature antifungal with a clinical update pattern dominated by topical routine use and safety-constrained systemic activity.
• The market is generic-driven; projections should assume pricing compression and focus on topical base retention plus systemic niche stabilization.
• Near-term trial activity is most likely supportive (equivalence, regimen confirmation, dermatology outcomes) rather than expansive new phase-3 antifungal discovery.
• Commercial outcomes will track label enforcement, competitor share shifts, and formulation mix rather than breakthrough clinical differentiation.

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FAQs

1) Is oral ketoconazole expected to recover to its historical growth rate?
No. Safety label restrictions constrain systemic adoption and keep systemic demand in a niche mode.

2) Which formulation drives the most resilient demand?
Topical ketoconazole, because it aligns with routine dermatology prescribing and favorable local safety.

3) What type of trials most commonly appear for ketoconazole today?
Bioequivalence, supportive safety/tolerability, and dermatology efficacy endpoint confirmation.

4) How does generic competition affect ketoconazole revenue projections?
It compresses pricing, so unit growth does not translate 1:1 into revenue growth.

5) What is the biggest commercial lever over the next forecast cycle?
Regulatory tightening or stabilization for systemic use, combined with competitive pressure in the azole class.

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References (APA)

[1] European Medicines Agency. (n.d.). Public assessment reports and product information for ketoconazole-containing medicines. https://www.ema.europa.eu
[2] U.S. Food and Drug Administration. (n.d.). Drug Safety Communications and product labeling for ketoconazole. https://www.fda.gov
[3] National Library of Medicine. (n.d.). ClinicalTrials.gov: Ketoconazole search results. https://clinicaltrials.gov