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Antifungal drug tested against brain tumors – trial stopped early

NCT ID NCT03796273

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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

Early phase 1

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

Participants

15 people

The number who actually took part.

Started

Mar 2019

Finished

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

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 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

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157, United States

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