UCSF BTC Clinical Trials

Maintenance Chemotherapy or Observation Following Induction Chemotherapy and Radiation Therapy in Treating Younger Patients With Newly Diagnosed Ependymoma


The purpose of this Phase III randomized trial is to study how well maintenance chemotherapy works in treating young patients with newly diagnosed ependymoma.

All patients in this trial will receive induction chemotherapy, which refers to chemotherapy as the initial, first-line treatment to make subsequent treatment (like radiation therapy) more effective. Chemotherapy drugs work in different ways to stop the growth of tumor cells, either by killing the cells or preventing them from dividing. This trial will use a variety of chemotherapy drugs in combination, which may kill more tumor cells.

Patients will then undergo 3D conformal radiation therapy (3DCRT), an advance radiation technique that customizes the shape and direction of multiple radiation beams, so that radiation is concentrated more precisely to a tumor while sparing healthy tissue around it.

Eligible participants in this trial will be randomized into two groups. After 3DCRT, one group will receive additional maintenance chemotherapy, involving a combination of chemotherapy drugs. The other group will remain under observation. By comparing these two groups, this study aims to determine how well maintenance chemotherapy works compared to observation, in patients with newly diagnosed ependymoma who have received induction chemotherapy and 3DCRT.  

  • Experimental Group: induction chemotherapy (vincristine sulfate, cyclophosphamide, and etoposide) + radiation therapy (3DCRT) + maintenance chemotherapy (liposomal vincristine sulfate, carboplatin, cyclophosphamide, etoposide, and cisplatin)
  • Active Comparator Group: induction chemotherapy (vincristine sulfate, cyclophosphamide, and etoposide) + radiation therapy (3DCRT) + observation


  1. Histologically confirmed intracranial ependymoma meeting the following criteria:
    1. Newly diagnosed disease
    2. Classic ependymoma (WHO II) or anaplastic ependymoma (WHO III), including the following subtypes:
      1. Clear cell
      2. Papillary
      3. Cellular
      4. Combination of the above
  2. No diagnosis of spinal cord ependymoma, myxopapillary ependymoma, subependymoma, ependymoblastoma, or mixed glioma
  3. Has undergone surgical resection of the primary tumor
    1. More than 1 attempted resection allowed
  4. No metastatic disease by MRI or cerebrospinal fluid (CSF) cytology
    1. CSR cytology from a ventriculostomy or permanent VP shunt that reveals the presence of tumor cells is indicative of metastatic disease
  5. No evidence of non-contiguous spread beyond the primary site as determined by pre- or post-operative MRI of brain, pre- or post-operative MRI of the spine, and post-operative CSF cytology obtained from the lumbar CSF space 
    1. Lumbar CSF examination may be waived if deemed to be medically contraindicated
  6. ECOG performance status (PS) 0-2
    1. Karnofsky PS for patients > 16 years of age
    2. Lansky PS for patients ≤ 16 years of age
  7. ANC ≥ 1,000/μL
  8. Platelet count ≥ 100,000/μL (transfusion independent)
  9. Creatinine clearance or radioisotope GFR ≥ 70 mL/min OR serum creatinine based on age/gender as follows:
    1. 0.4 mg/dL (1 month to < 6 months of age)
    2. 0.5 mg/dL (6 months to < 1 year of age)
    3. 0.6 mg/dL (1 to 2 years of age)
    4. 0.8 mg/dL (2 to < 6 years of age)
    5. 1.0 mg/dL (6 to 10 years of age)
    6. 1.2 mg/dL (10 to 13 years of age)
    7. 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to < 16 years of age)
    8. 1.7 mg/dL (male) or 1.4 mg/dL (female) (≥ 16 years of age)
  10. Total bilirubin ≤ 1.5 times upper limit of normal (ULN) (≤ 3 times ULN for patients with Gilbert syndrome or hemolytic anemia)
  11. AST or ALT < 3 times ULN
  12. Adequate cardiac function defined as 1 of the following:
    1. Shortening fraction ≥ 27% by ECHO
    2. Ejection fraction ≥ 50% by gated radionuclide study.
  13. Not pregnant or nursing
    1. Patients who agree to stop nursing while on this study are allowed
  14. Negative pregnancy test
  15. Fertile patients must use effective contraception
  16. No prior treatment for ependymoma other than surgical intervention and corticosteroids

For the most up-to-date list of criteria, please visit clinicaltrials.gov.


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