Phase 1 N=3 Treatment

Topotecan by Intracerebral Clysis for Recurrent Primary Malignant Brain Tumors

Brain Neoplasms, Primary Malignant

Bottom Line

View on ClinicalTrials.gov: NCT00308165 ↗

Enrolled (actual)

Serious AEs

12.5%

Results posted

Jun 2025

Primary outcome: Primary: Number of Participants With Dose Limiting Toxicities — 0; 0; 0; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 1
Interventions: Convection-Enhanced Delivery (Procedure); Topotecan (Drug)
Age: Pediatric, Adult, Older Adult · 1+ yrs
Sex: All
Sponsor: Jeffrey N. Bruce
Primary completion: Mar 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Dose Limiting Toxicities	0; 0; 0; 0; 2	—
PRIMARY Maximum Tolerated Dose (MTD)	0.1	—
SECONDARY Time to Tumor Progression/Recurrence	70.5; 6.7; 31.7; 36.7; 15.0	—
SECONDARY Time to Death	80; 46; 54; 35.7; 27.3	—

Summary

This study will evaluate the safety and efficacy of a chemotherapeutic drug (topotecan) as it is given directly into brain tumors by a delivery technique called convection-enhanced delivery. This drug has been used for different types of cancer, but in this study it will be given by an experimental delivery technique designed to maximize the amount of drug delivered to the brain tumor and minimize the side effects in other parts of the body. This study will also evaluate advanced magnetic resonance (MR) imaging techniques. The study will assess quality of life parameters throughout the follow-up period.

Eligibility Criteria

Inclusion Criteria

Patients with primary malignant brain tumor, or a newly diagnosed or recurrent malignant tumor of the brainstem. Patients with tumors of the brain must have been previously treated with external beam radiation. Patients with brainstem gliomas may or may not have been previously treated.
Patients with a tumor that is stereotactically accessible (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 100 cc in total volume).
Patients who demonstrate evidence of increasing contrast enhancement on MR or CT imaging while on stable or increasing dose of steroid.
Patients with a Karnofsky Performance Score of greater than or equal to 60.
Men and women of child-bearing potential must practice birth control. Women of child bearing potential must have a negative serum or urine pregnancy test within 7 days of study entry.
Patients must possess the ability to give Informed Consent. Parent or guardian may give informed consent for minors.
Patients must be willing to and medically capable of undergoing the surgical operation.
Patients may not be receiving other investigational agents for the treatment of malignant astrocytoma.
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 1 year old to participate in the study.

Exclusion Criteria

Patients with diffuse subependymal or cerebrospinal fluid (CSF) disease.
Patients with tumors involving the cerebellum, or both hemispheres.
Patients with an active infection requiring treatment or having an unexplained febrile illness.
Patients who are known HIV positive or who are known positive for Hepatitis B or C virus
Patients who have received any form of radiation or chemotherapy within 4 weeks of protocol enrollment.
Patients with systemic diseases which may be associated with unacceptable anesthetic/operative risk.
Patients who have previously received systemic topotecan for their tumor
Patients less than 1 year of age
Patients who are not able to receive an MRI scan

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00308165). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.