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Antifungal drug tested against brain cancer — but trial stops early

NCT ID NCT04869449

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial tested whether ketoconazole, a drug normally used for fungal infections, can enter brain tumors in high enough amounts to stop tumor cells from dividing. Only 1 person was enrolled before the study was terminated. The goal was to measure drug levels in the tumor and check safety, but no conclusions can be drawn from such a small, incomplete study.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ketoconazole (an antifungal drug)
What this could lead to
If successful, this could show whether ketoconazole reaches brain tumors in high enough levels to be studied further as a possible treatment.
What could go wrong
This was a very early phase 0 trial with only 1 participant and was terminated early, so results are extremely limited and may not apply to others.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

1 person

The number who actually took part.

Started

May 2022

Finished

May 2022

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Age ≥18 years * Evidence of primary or recurrent high-grade gliomas (HGG) that in the opinion of the treating team would require surgical resection * Karnofsky Performance Score (KPS) ≥ 60% * Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 * Life expectancy greater than 12 weeks * Adequate liver function defined as Alanine aminotransferase (ALT),Aspartate transaminase (AST), Alkaline phosphatase (ALP) within 1.5x institutional upper limit of normal * Adequate renal function defined as estimated glomerular filtration rate (eGFR) levels within 1.5x the institutional upper limit of normal * Ability to swallow medication * Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) for the duration of study participation. * Ability to understand and willingness to sign a written informed consent document * Be able to comply with treatment plan, study procedures and follow-up examinations Exclusion Criteria: * Patients may not be receiving any other investigational agents while on study * Patients who have known allergy to ketoconazole or other azoles * Patients who have previously had a severe side effect, such as agranulocytosis and neutropenia, in conjunction with previous azole class drugs for a parasitic infection * Patients with a history of acute or chronic hepatitis * Patients with liver enzymes (ALT, AST, ALP) \>1.5x above normal range for the laboratory performing the test * Patients who are taking metronidazole and cannot be safely moved to a different antibiotic greater than 7 days prior to starting ketoconazole therapy * Patients who are taking any anti-convulsant medication that interferes with the cytochrome P450 pathway (e.g. phenytoin, phenobarbital, carbamazepine, etc.) and who cannot be switched to alternative medications such as keppra (levetiracetam) * Uncontrolled intercurrent illness such as chronic hepatitis, acute hepatitis, or psychiatric illness/social situation that would limit compliance with study requirements * Patients with a history of Addison's disease or other forms of adrenal insufficiency * Patient with little or no stomach acid production (achlorhydria) * Pregnant and breast feeding women * Patients with a history of any medical or psychiatric condition or laboratory abnormality that in the opinion of the investigator may increase the risks associated with the study participation or investigational product administration or may interfere with the interpretation of the results. * Patients who are not available for follow-up assessments or unable to comply with study requirements. * Patients who are currently taking medications that induce the metabolism of ketoconazole, such as isoniazid, nevirapine, rifamycins (such as rifabutin, rifampin), or St. John's wort and cannot be safely discontinued off of them for the duration of the trial. * Patients who are currently taking medications for which the metabolism may be affected by ketoconazole, which include but are not limited to: benzodiazepines (such as alprazolam, midazolam, triazolam), domperidone, eletriptan, eplerenone, ergot drugs (such as ergotamine), nisoldipine, drugs used to treat erectile dysfunction (ED) or pulmonary hypertension (such as sildenafil, tadalafil), some drugs used to treat seizures (such as carbamazepine, phenytoin), some statin drugs (such as atorvastatin, lovastatin, simvastatin). * Patients who are non-English speakers * Patients who are not capable of understanding the consent form and would need a legally authorized representative.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Penn State Milton S Hershey Medical Center

    Hershey, Pennsylvania, 17033, United States

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Other studies related to the condition(s) this trial covers.