Infusion of 5-Azacytidine (5-AZA) Into the Fourth Ventricle in Children With Recurrent Posterior Fossa Ependymoma
Condition: Brain Tumor Recurrent
MeSH terms: Ependymoma; Recurrence
Study type: Interventional
Phase: Early Phase 1

Summary: 
      The goal of this clinical research study is to establish the safety of direct administration
      of 5-Azacytidine into the fourth ventricle of the brain or resection cavity in patients with
      recurrent posterior fossa ependymoma.
    

Detailed description: 
      If the participant is eligible to take part in this study, the participant will have surgery
      to place a catheter into the Ommaya reservoir. The Ommaya reservoir is a catheter system that
      allows drugs to be administered directly to parts of the brain. This catheter will used for
      the infusion of 5-Azacytidine directly into the 4th ventricle of the brain, which is 1 or the
      4 connected fluid-filled cavities in the brain.

      If the study doctor thinks it is necessary, based on the location of the tumor, the tumor may
      also be removed while the participant is already under anesthesia just before the catheter is
      placed.

      Study Drug Administration:

      The participant will receive 12 weekly (+/- one day) 10 mg infusions of 5-Azacytidine.

      5-Azacytidine will be infused through the Ommaya reservoir catheter directly into the 4th
      ventricle of the brain starting at a minimum of 7 days after the catheter placement surgery.
      A MRI will be done to confirm adequate cerebrospinal fluid flow. The 5-Azacytidine infusion
      will last 2-3 minutes.

      If the participant already has an Ommaya catheter, 5-Azacytidine will begin after an MRI has
      confirmed adequate cerebrospinal fluid flow.

      Study Visits:

      Prior to first infusion:

      Medical history will be reviewed and any updates to health will be recorded. Physical and
      Neurological exam with vital signs will be done. Blood (about 1 teaspoon) will be drawn for
      routine test. A lumbar puncture will be done. A MRI scan of the brain and spine will be done
      to check the status of the disease.

      On the days of the 5-Azacytidine Infusion:

      A neurological exam with vital signs will be done. A Ommaya reservoir tap (a catheter is
      placed into the Ommaya reservoir to give the 5-Azacytidine infusion.

      Cerebrospinal fluid (about 1 teaspoon) will be collected for routine tests.

      Within 7 days of completing the Final infusion:

      A neurological exam with vital signs will be done. A lumbar puncture will be done. A MRI scan
      of the brain and spine will be done to check status of the disease.

      Length of Study:

      The participant will receive 12 infusions of 5-Azacytidine, as long as the doctor thinks it
      is in their best interest. The participant will no longer be able to receive the study drug
      if the disease gets worse, if intolerable side effects occur, or if unable to follow study
      directions.

      This is an investigation study. 5-Azacytidine is FDA approved and commercially available to
      be given subcutaneous or into the bloodstream (intravenously) but has never been given in the
      4th ventricle of the brain. The infusion of 5-Azacytidine into the 4th ventricle of the brain
      is investigational.

      Up to 10 patients will be enrolled in this study. All will be enrolled at Children's Memorial
      Hermann Hospital
    

Intervention name(s): 5-Azacytidine
Intervention type(s): Drug
Intervention description(s): 5-Azacytidine 10 mg into the fourth ventricle of the brain via the Ommaya reservoir 1 day a week for 12 weeks.
Other intervention name(s): Vidaza, Mylosar
Intervention MeSH terms: Azacitidine
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Ependymoma