Antifungal drug tested against brain cancer — but trial stops early
NCT ID NCT04869449
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 Hide 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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Glioblastoma are added.
Genom att skicka in godkänner du våra Användarvillkor
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a trio of repurposed drugs shrink recurrent brain tumors?
- Can an existing drug boost bevacizumab against recurrent brain cancer?
- One extra radiation dose after standard brain cancer therapy: can it boost the immune system?
- Radioactive tracer lights up Oxygen-Starved brain tumors
- Can a common virus vaccine help fight brain cancer?
- Radioactive tiles target brain tumors in frail patients