Phase II Study of Intraventricular Methotrexate in Children With Recurrent or Progressive Malignant Brain Tumors

January 7, 2020 updated by: Ziad Khatib, Nicklaus Children's Hospital f/k/a Miami Children's Hospital

A Phase II Study of Intraventricular Methotrexate With Systemic Topotecan and Cyclophosphamide in Children With Recurrent or Progressive Malignant Brain Tumors

The purpose of this research study is to test an experimental treatment method for recurrent or progressive brain tumors in children aged from 0-22 years. The use of methotrexate and chemotherapy (topotecan and cyclophosphamide) is experimental in this study. This means that their use by themselves or together has not been approved by the U.S. Food and Drug Administration for this usage.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

The purpose of this research study is to test an experimental treatment method for recurrent or progressive brain tumors in children aged from 0-22 years. The use of methotrexate and chemotherapy (topotecan and cyclophosphamide) is experimental in this study. This means that their use by themselves or together has not been approved by the U.S. Food and Drug Administration for this usage. This study will attempt to determine via serial MRI scans if methotrexate administration into the lateral or fourth ventricle in combination with systemic intravenous topotecan and cyclophosphamide is effective in reducing tumor burden in the brain and spine for patients with recurrent or progressive brain tumors, including medulloblastoma, ependymoma, PNET (Primitive Neuroectodermal Tumor), atypical teratoid/rhabdoid tumor (AT/RT), and other malignant embryonal tumors. In addition, the study will evaluate the toxicity of the above mentioned experimental treatment during a two-year progression-free survival and survival of children with recurrent malignant brain tumors.

Study Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33155
        • Nicklaus Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 22 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Recurrent or progressive supratentorial or posterior fossa tumors with measurable disease on imaging studies of the brain and spine or CSF cytology.
  • Tumor histology, including: Medulloblastoma , PNET, or other embryonal tumor, Atypical teratoid/rhaboid tumor (AT/RT), and Ependymoma.
  • Leptomeningeal dissemination of a previously diagnosed CNS tumor.
  • Diagnosis of tumor pathology will be based upon pathology diagnosis from previous surgeries for patients with recurrent tumors.
  • Patients must have received prior radiation before current recurrence, unless the patient is less than 36 months old at diagnosis and has progressed after at least one upfront chemotherapy regimen in which case no prior radiation is required.
  • Patients must have a life expectancy of at least 12 weeks as indicated by the patient's oncologist and/or neurosurgeon.
  • Lansky or Karnofsky Performance status of at least 50.
  • Negative pregnancy test.
  • Specific organ function requirements for: Central Nervous System, Bone Marrow, renal and liver.

Exclusion Criteria:

  • Patients that do not meet the inclusion criteria above.
  • Pregnant or lactating female patients.
  • Patients currently enrolled in another experimental treatment protocol.
  • Patients with documented allergies to any of the chemotherapy agents used in this study.
  • Patient/Parent refuses study participation.
  • Patient is severely somnolent or comatose.
  • Unable or unwilling to commit to return or to follow-up visits.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Intra thecal methotrexate
IT methotrexate via Ommaya reservoir with concomitant systemic topotecan and cyclophosphamide
Drug: Intra thecal methotrexate
IT methotrexate via Ommaya reservoir with concomitant systemic topotecan and cyclophosphamide
Other Names:
  • Intra thecal methotrexate, topotecan and cyclophosphamide
Drug: topotecan
To be administered in conjunction with methotrexate
Other Names:
  • Intra thecal methotrexate, topotecan and cyclophosphamide
Drug: cyclophosphamide
To be administered in conjunction with methotrexate and topotecan
Other Names:
  • Intra thecal methotrexate, topotecan and cyclophosphamide

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Tumor response
Time Frame: 6 months
MRI response
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Toxicity of chemotherapy regimen (topotecan and cyclophosphamide)
Time Frame: 6 months
Laboratory results, MRI, physical examination
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Investigators

  • Principal Investigator: Ziad Khatib, MD, Nicklaus Children's Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 1, 2016

Primary Completion (Actual)

December 9, 2019

Study Completion (Actual)

December 9, 2019

Study Registration Dates

First Submitted

February 11, 2016

First Submitted That Met QC Criteria

February 11, 2016

First Posted (Estimate)

February 17, 2016

Study Record Updates

Last Update Posted (Actual)

January 10, 2020

Last Update Submitted That Met QC Criteria

January 7, 2020

Last Verified

January 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NCH-CNS-1601

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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