Methotrexate, Cyclophosphamide & Topotecan

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




The main purpose of this study is to determine if infusions of methotrexate into the fourth ventricle of the brain in combination with IV administration of topotecan and cyclophosphamide is effective in reducing disease in the brain and spine for patients with recurrent or progressive brain tumors. These tumors include medulloblastoma, ependymoma, PNET, atypical teratoid/rhabdoid tumor or other malignant brain tumor with recurrence or progression. 


What to expect:

  • Prior to beginning study treatment, patients will have surgery to place an Ommaya reservoir (special catheter to deliver the study treatment to the brain). 





  1. Methotrexate: Patients will receive methotrexate through the Ommaya reservoir.

  2. Topotecan: Patients will receive toptecan intravenously (IV).

  3. Cyclophosphamide: Patients will receive cyclophosphamide intravenously (IV).


Inclusion Criteria

  • Patient’s must be 0-22 years of age at the time of recurrence or progression

  • Patients with recurrent or progressive medulloblastoma, ependymoma, or atypical teratoid/rhabdoid tumor or other malignant brain tumor with measurable disease on imaging scans of the brain and spine or in the cerebral spinal fluid 

  • Leptomeningeal dissemination of a previously diagnosed CNS tumor.

  • Patent must have Ommaya catheter or agree to have one placed

  • Patients must have received prior radiation before current recurrence (if the patient is less than 36 months old at diagnosis and has progressed after at least one chemotherapy regimen, no prior radiation is required). 

  • Life expectancy of at least 12 weeks

  • Patients must have adequate organ function that will be confirmed through a routine blood draw


Exclusion Criteria

  • Currently enrolled on another treatment trial 

  • Documented allergic reaction to any of the drugs used in this study 

  • Pregnant or lactating women 

  • Patient is severely lethargic (somnolent) or in a coma


Full Details on ClinicalTrials.Gov

This protocol is only available at the available at the Nicklaus Children’s Hospital (Miami, FL).



Principal Investigator: Ziad Khatib, MD

Phone: (305) 662-8360


Study Contact: Jenny Esteves

Phone: (786) 624-2854


Hope4ATRT Foundation

is a research entity of

Out of Zion, Inc.

Mailing Address: 

P.O. Box 6130   New York, NY  10150