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.

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
Neoplasms	6
Dermatitis	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
Florida	17
Pennsylvania	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
Pfizer	8

Sponsor Type

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

Last updated: July 27, 2026

Ketoconazole Clinical Trials Update, Market Analysis, and Revenue Projection (2026–2036)

Ketoconazole’s clinical pipeline is narrow and largely legacy-driven. Market trajectory is shaped by (1) formulation life-cycle management (topical and oral), (2) safety-driven utilization changes after oral hepatotoxicity warnings, and (3) competitive pressure from newer antifungals in most indications. No current, specific late-stage global development program dominates the asset category at a level that supports confident, drug-level forecasting without a named formulation, sponsor, route (oral vs topical), and branded vs generic status.

Is ketoconazole still in clinical trials, and what is the latest update?

Bottom line: Ketoconazole is present in the literature and in smaller studies, but the category does not show a clear, singular “active” late-stage registrational program that would materially change the drug-level market outlook in the way typical with blockbuster pipelines.

What types of ketoconazole studies are still happening

• Topical formulation optimization (vehicle, penetration enhancers, stability)
• Dermatology indication support (tinea versicolor, seborrheic dermatitis, superficial fungal infections)
• Alternative dosing and regimen studies within legacy guidance
• Comparative trials versus other azoles and non-azole classes (commonly in mild-to-moderate skin disease)

Where ketoconazole trial activity is most likely to surface

• Dermatology networks running investigator-led or generic-development comparatives
• Formulation bridging rather than new clinical endpoints

What phase-level signals matter for market projections

For market-impacting forecasts, the relevant signals are:

• Phase 3 or confirmatory studies tied to new label indications
• New competitive delivery systems that support premium pricing
• Secure FDA approval pathway for a new dosage form that changes substitution patterns

As a category, ketoconazole has more “formulation maintenance” and less “label expansion” momentum.

Which ketoconazole indications have the most clinical and commercial traction today?

Bottom line: Commercial traction is route- and indication-dependent. Topical use sustains more consistent demand than oral, which faces stronger safety and prescribing constraints.

Indication clustering (commercially relevant)

• Seborrheic dermatitis / dandruff (topical)
• Tinea versicolor (topical and oral in some settings)
• Superficial fungal infections (topical)
• Off-label or specialty settings (route-dependent; varies by geography)
• Endemic mycoses and systemic fungal disease have historical use but weaker current positioning versus newer agents, depending on country formularies

What is ketoconazole’s market size, by route and formulation?

Bottom line: Ketoconazole market is primarily generic and off-patent across most geographies, with demand anchored in topical dermatology and legacy oral use where permitted. Category-level market expansion is typically driven by population, dermatology prevalence, and growth of retail generics, not by premium innovation.

Commercial demand drivers

• High prevalence of seborrheic dermatitis and superficial fungal infections
• Continued OTC-like channels in certain markets for dandruff-related indications (where applicable)
• Competitive substitution by newer azoles and allylamines depending on formulary and pricing

Commercial headwinds

• Oral ketoconazole’s positioning is constrained by safety risk management, which reduces prescriber confidence and limits uptake
• Therapeutic switching toward agents with better safety profiles

How does ketoconazole compare with newer antifungals on efficacy and safety?

Bottom line: Ketoconazole’s relative advantage is usually price and availability rather than a clear efficacy superiority in modern practice. In systemic settings, prescribers commonly prefer agents with more favorable safety profiles.

Competitive comparison set (practical substitution)

• Other azoles: fluconazole, itraconazole, voriconazole, posaconazole
• Topical competitors: terbinafine, clotrimazole, ciclopirox, etc. depending on indication
• Systemic practice: newer agents often dominate formulary decisions for invasive disease

What is the Orange Book status of ketoconazole products?

No complete, product-level Orange Book mapping can be produced here without specifying:

• country (US vs others),
• active ingredient salt/form,
• and a particular marketed product name (brand or NDA/ANDA holder).

Under standard Orange Book practice, ketoconazole products in the US are largely generic with broad expiration histories, but drug-level “status” tables require exact application numbers.

What patents protect ketoconazole formulations, and do they affect generic competition?

Bottom line: Ketoconazole is widely off-patent in most major markets. Patent leverage, where present, is typically tied to:

• specific formulation compositions,
• specific delivery systems or vehicles,
• and sometimes method-of-use claims.

For market projections, the key consequence is that generic competition is typically established, so near-term pricing tends toward generics-driven erosion rather than innovation-driven growth.

What generic entry risks exist for ketoconazole?

Bottom line: In most markets, the risk is less about whether generics can enter and more about market share stability amid ongoing generics manufacturing competition and substitution by alternative antifungals.

Typical generic dynamics

• Price compression after multiple ANDAs/locals enter
• Supply stability issues affecting tender outcomes in some geographies
• Formulation-level substitution if patients or clinicians prefer certain vehicles

What do current clinical trial registries show for ketoconazole’s ongoing development?

Bottom line: The observable ketoconazole trial footprint is more consistent with:

• small comparative or formulation studies, and
• registry-listed observational or supportive studies,

than with large, late-stage, label-changing registrational programs.

When does ketoconazole lose exclusivity?

Bottom line: Ketoconazole’s major market authorizations are, by practical industry standards, already beyond primary exclusivity in most jurisdictions for common dosage forms. Exclusivity-driven forecasts are therefore more useful for specific, still-protected new formulations rather than ketoconazole as a category.

How to project ketoconazole revenue: scenario framework (2026–2036)

Bottom line: Without a specified formulation, route, and marketed product reference point, any “drug-level” forecast risks being category-incorrect. The most decision-useful approach is scenario-based projection for the ketoconazole market category by route.

Projection structure used for antifungals in off-patent categories

1. Volume: dermatology prevalence, recurrence patterns, and population growth
2. Price: generics price erosion and channel mix changes
3. Share shift: substitution toward newer antifungals
4. Regulatory and safety: oral prescribing constraints and risk communications

Revenue projection (category-level, directional)

• Topical ketoconazole: low-to-mid single digit CAGR is plausible in many markets due to steady dermatology use and generic affordability, partially offset by substitution by other topicals.
• Oral ketoconazole: slower growth or flat-to-declining trajectory in many markets due to safety-driven prescribing and competition from other systemic azoles.

Key sensitivities that swing forecasts

• Restriction tightening or re-interpretation of oral risk communications
• Reimbursement/formulary actions for dermatology indications
• Competitive pricing from other generic azoles and allylamines
• Availability and manufacturing capacity in peak-demand periods (tender cycles)

Which companies commercialize ketoconazole, and how does the competitive landscape affect pricing?

Bottom line: Ketoconazole is competitive and fragmented. Pricing trends are typically driven by:

• number of generic manufacturers per country,
• tender practices,
• and distributor bargaining power.

For an investable view, the relevant market intelligence is brand vs generic mix in each country and tender-based procurement share.

What clinical outcomes matter most for ketoconazole adoption?

For topical fungal dermatology, outcomes that influence continued use and share include:

• lesion clearance rates,
• time-to-symptom relief (itching, erythema, scaling),
• recurrence rates or need for retreatment,
• tolerability and local irritation rates.

For oral use, outcomes include:

• symptom resolution in the labeled or permitted off-label setting,
• liver function monitoring adherence,
• safety incident rates affecting prescribing behavior.

Key Takeaways

• Ketoconazole’s market outlook is shaped by route and safety-driven oral constraints, with topical use providing the most stable demand.
• The clinical pipeline for ketoconazole does not show clear evidence of a dominant, late-stage, label-changing program that would reset the category’s trajectory.
• Revenue growth in most scenarios is driven by volume expansion and channel mix, while pricing remains pressured by generic competition and substitution.

FAQs

1. What is the most common ketoconazole use case today (topical vs oral)?
2. Do newer azoles reduce ketoconazole prescribing for seborrheic dermatitis?
3. Are there any current large Phase 3 ketoconazole trials for new indications?
4. How does oral ketoconazole safety risk management affect market demand by country?
5. What formulation types (cream, shampoo, gel) drive ketoconazole market share most?

References

1. U.S. FDA. Drug Safety Communication on ketoconazole oral products. (FDA website).
2. EU European Medicines Agency. Summary of product characteristics and safety communications for ketoconazole-containing medicines. (EMA website).
3. ClinicalTrials.gov. Search results for “ketoconazole” (retrieved via the database).