NCT00308165

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Mar 2004

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2004

Completed
2.1 years until next milestone

First Submitted

Initial submission to the registry

March 27, 2006

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 29, 2006

Completed
8.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2015

Completed
10 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2016

Completed
9.5 years until next milestone

Results Posted

Study results publicly available

June 27, 2025

Completed
Last Updated

June 27, 2025

Status Verified

June 1, 2025

Enrollment Period

11 years

First QC Date

March 27, 2006

Results QC Date

February 3, 2018

Last Update Submit

June 11, 2025

Conditions

Brain Neoplasms, Primary Malignant

Keywords

Recurrent Primary Malignant Brain TumorsBrain TumorsNeoplasms, BrainBrain CancerBrain cancer treatmentTopotecan

Outcome Measures

Primary Outcomes (2)

  • Number of Participants With Dose Limiting Toxicities

    During treatment, up to 5 Days

  • Maximum Tolerated Dose (MTD)

    If 2 participants within a cohort develop dose-limiting toxicity (DLT), the prior dose level is considered the maximum tolerated dose (MTD). Dose-Limiting Toxicities (DLT) will be defined as any new (or increased from baseline) treatment-related (drug and/or device) neurological deficits as exhibited on neurological examination with severity of grade 3 or higher.

    During treatment, up to 5 Days

Secondary Outcomes (2)

  • Time to Tumor Progression/Recurrence

    Treatment to progression, Up to 8 years

  • Time to Death

    Treatment to Time of Death, Up to 8 Years

Study Arms (1)

Topotecan

EXPERIMENTAL

Once a plastic catheter is placed, within 24 hours of placement, the catheter will be connected to a small pump at the bedside, and the convection-enhanced delivery of the Topotecan will begin. The Topotecan will be infused for 4 to 5 days after which time the catheters will simply be pulled out. Patients will be monitored with blood tests and MRI scans during the treatment and at different time periods during the following months.

Procedure: Convection-Enhanced DeliveryDrug: Topotecan

Interventions

Convection-Enhanced DeliveryPROCEDURE

microinfusion pumps to deliver chemotherapy directly into brain tumors

Also known as: Intracerebral Clysis
Topotecan
TopotecanDRUG

chemotherapeutic drug for the treatment of brain tumors

Also known as: Hycamtin
Topotecan

Eligibility Criteria

Age1 Year+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Columbia University Medical Center Neurological Institute

New York, New York, 10032, United States

Location

Related Publications (1)

  • Anderson RC, Kennedy B, Yanes CL, Garvin J, Needle M, Canoll P, Feldstein NA, Bruce JN. Convection-enhanced delivery of topotecan into diffuse intrinsic brainstem tumors in children. J Neurosurg Pediatr. 2013 Mar;11(3):289-95. doi: 10.3171/2012.10.PEDS12142. Epub 2012 Dec 14.

    PMID: 23240851DERIVED

MeSH Terms

Conditions

Brain Neoplasms

Interventions

Topotecan

Condition Hierarchy (Ancestors)

Central Nervous System NeoplasmsNervous System NeoplasmsNeoplasms by SiteNeoplasmsBrain DiseasesCentral Nervous System DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

CamptothecinAlkaloidsHeterocyclic Compounds

Results Point of Contact

Title
Jeffrey N. Bruce, MD
Organization
Columbia University
jnb2@columbia.edu
212-305-7346

Study Officials

  • Jeffrey Bruce, MD

    Columbia University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Edgar M. Housepian Professor of Neurological Surgery Research

Study Record Dates

First Submitted

March 27, 2006

First Posted

March 29, 2006

Study Start

March 1, 2004

Primary Completion

March 1, 2015

Study Completion

January 1, 2016

Last Updated

June 27, 2025

Results First Posted

June 27, 2025

Record last verified: 2025-06

Locations

US(1)