Brain tumor chemo delivered by pump shows promise in tiny safety trial
NCT ID NCT03154996
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase study tested whether pumping the chemotherapy drug topotecan directly into the brain is safe for people with recurrent high-grade gliomas (aggressive brain tumors). Only 5 patients took part, and the main goal was to find a safe dose. The drug was delivered continuously through a small pump implanted under the skin. While the trial is too small to say if it works, it lays groundwork for future studies on this direct-delivery approach.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Topotecan (a chemotherapy drug)
- What this could lead to
- If it works, this could point toward a new way to deliver chemotherapy directly to brain tumors, potentially improving treatment for recurrent gliomas.
- What could go wrong
- This is a very early, tiny trial (only 5 people) focused on safety, not effectiveness. The drug is delivered directly into the brain, which carries risks like infection or brain injury.
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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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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5 people
The number who actually took part.
- Started
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Jan 2018
- Finished
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Aug 2021
- 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
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18 years and older
- Sex
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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: * Patients must have a recurrent malignant glioma (World Health Organization (WHO) grade III-IV), including recurrent glioblastoma, anaplastic astrocytoma, anaplastic oligodendroglioma, anaplastic oligoastrocytoma, and anaplastic ependymoma. Stereotactic biopsies will be performed to confirm this diagnosis prior to initiating the treatment. * Patients with tumors of the brain must have been previously treated with surgical resection, external beam radiation, and temozolomide chemotherapy. * An magnetic resonance (MR) scan must be obtained within 30 days of enrollment and must demonstrate an enhancing mass without significant mass effect. Tumors must be less than 32 cc in total volume. The lesion must be stereotactically accessible. * Patients must have demonstrated evidence of increasing contrast enhancement on MR or computed tomography (CT) imaging while on stable or increasing dose of steroid. * Karnofsky performance score of greater than or equal to 60. * Men and women of childbearing potential must practice birth control. Women of child bearing potential must have a urine pregnancy test within 7 days of study entry. * Patients must possess the ability to give Informed Consent. * Patients must be willing to and medically capable of undergoing the surgical operation. * There is no upper age limit. Patients at extreme upper end of the age spectrum will not be automatically excluded, but will be carefully scrutinized to determine their suitability for this procedure. * Patients must be at least 18 years old. * Patients must have normal organ and marrow function as defined below: * Leukocytes: ≥3,000/ cells per microliter of blood (mcL) * Absolute neutrophil count: ≥1,500/mcL * Platelets: ≥100,000/mcL * Total bilirubin: within normal institutional limits * aspartate aminotransferase (AST) (SGOT)/alanine aminotransferase (ALT) (SGPT): ≤2.5 × institutional upper limit of normal * Creatinine: within normal institutional limits OR * Creatinine clearance: ≥60 mL/min/1.73 m2 for patients with creatinine levels above institutional normal. Exclusion Criteria: * Patients with diffuse subependymal or cerebral spinal fluid (CSF) disease. * Patients with tumors involving the cerebellum or both cerebral hemispheres. * Patients with an active infection requiring treatment or having an unexplained febrile illness. * Patients who are known HIV, Hepatitis B or Hepatitis C positive. HIV-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with topotecan. In addition, these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated. * Patients with systemic diseases which may be associated with unacceptable anesthetic/operative risk. * Patients who have previously received systemic topotecan for their tumor * Patients who are not able to receive an MRI scan. * History of allergic reactions attributed to compounds of similar chemical or biologic composition to topotecan, other topoisomerase inhibitors or gadolinium compounds.
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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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Columbia University Irving Medical Center
New York, New York, 10032, United States
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