Antifungal drug tested against brain tumors – trial stopped early
NCT ID NCT03796273
First seen Jun 25, 2026 · Last updated Jul 31, 2026 · Updated 2 times
Summary
This early-phase trial tested whether the antifungal drug ketoconazole could block a protein called tGLI1 in brain tumors. It included 15 people with breast cancer that spread to the brain or with a type of brain cancer called glioma. The study was terminated early, so we have limited information on whether the drug worked or was safe.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ketoconazole
- What this could lead to
- If it had worked, this could have pointed toward a new way to treat brain tumors by targeting a specific protein.
- What could go wrong
- This was a very early, small trial that was terminated before completion, so we don't have enough data to know if it works or is safe.
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
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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15 people
The number who actually took part.
- Started
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Mar 2019
- Finished
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Oct 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
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Anyone
- Healthy volunteers
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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 greater than or equal to 18 years old Subjects must have a history of: * Histologically confirmed primary breast cancer including primary invasive and metastatic breast cancers with imaging findings consistent with brain metastasis. In the event that a patient presents with an initial diagnosis of metastatic breast cancer with imaging findings of a new brain metastases and unequivocal imaging findings of a primary breast cancer, consideration for study enrollment requires approval from the study chair (primary cohort) OR * Histologically confirmed primary glioma including astrocytoma or oligodendroglioma of any World Health Organization grade with imaging findings consistent with recurrent or progressive disease (exploratory cohort). Patients with ependymoma will not be included. * Subjects must be undergoing surgical resection for clinical purposes with anticipated resection of at least 300 mg of tissue. * Patients with any prior number of radiation (including brain radiation), chemotherapy, or surgical interventions will be eligible for this protocol. * The effects of ketoconazole on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. * Ability to understand and the willingness to sign an Institutional Review Board (IRB)-approved informed consent document (either directly or via a legally authorized representative). Exclusion Criteria: * Subjects with contraindication to ketoconazole including: * Prior allergic reaction or intolerance of ketoconazole * Known active hepatitis * QTc prolongation (based on electrocardiography \[EKG\] obtained within 21 days of enrollment, with a threshold of \>450 ms in males and \>470 ms in female) * Known liver cirrhosis will be excluded from enrollment * Positive serum pregnancy test within 21 days of enrollment * Subjects currently taking medications that are included in the contraindicated concurrent medications section of the Food and Drug Administration (FDA) approved indications for ketoconazole will be required to complete a seven day wash out period prior to consideration for enrollment. * Subjects for whom collection of blood, or tissue samples is unsafe or clinically inadvisable. * Pregnant women are excluded from this study because ketoconazole is a Class B agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with ketoconazole, breastfeeding should be discontinued if the mother is treated with ketoconazole.
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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
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Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157, United States
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