Clinical Research Study Viewer

ACNS0831 Phase III Randomized Trial of Post-Radiation Chemotherapy in Patients with Newly Diagnosed Ependymoma Ages 1 to 21 years

Purpose of study:

Recent studies of young children have shown that chemotherapy (anti-cancer drug therapy) can help shrink or get rid of ependymoma tumors. Other studies have shown that some children (about 15%) who received chemotherapy before radiation therapy had their tumors grow while receiving the chemotherapy. For this reason, study doctors think that if people with ependymoma are to receive chemotherapy before radiation therapy, the chemotherapy should not last a long time. This is called a short course of chemotherapy.

Since your tumor was partially removed by surgery, you will receive induction chemotherapy while on this study and, depending on your response to the induction chemotherapy, one or more of the following: a second surgery, radiation therapy, maintenance chemotherapy, and/or observation.

The overall goals of this part of the study are:
For patients whose tumor was partially removed by surgery, to see if treatment with a short course of chemotherapy can:
a) kill any remaining tumor, or
b) increase the number of patients whose remaining tumor can be completely removed by a second surgery.

Other goals of this study are to:

  • Study the effects of surgery, radiation therapy and chemotherapy on learning, thinking, hearing and the production of hormones (substances made in the brain that affect growth and development).
  • Study tumor tissue and blood for possible genetic and biologic factors related to ependymoma.

Who can participate:

Patients must be greater than 12 months of age and less than 21 years of age at the time of study enrollment.

Patients must be newly diagnosed with intracranial ependymoma. Patients with classic ependymoma (WHO II) or anaplastic ependymoma (WHO III) are eligible, as are various subtypes described as clear cell, papillary, cellular or a combination of the above.

The diagnosis of intracranial ependymoma must be histologically confirmed.

Additional screening for eligibility will be required.

What will happen during the study:


All subjects will begin with a short course of chemotherapy, called Induction chemotherapy, which will start 2 weeks after entering the study. The chemotherapy will be given over 7 weeks. You will be evaluated (have scans) after the completion of Induction chemotherapy. What happens next is determined by the features of your tumor and how your tumor reacted to Induction chemotherapy.

  • If the tumor has spread into other areas of the brain (metastasized), you will be taken off this study, and your doctors will talk to you about other treatment options.
  • If the tumor has completely gone away after Induction chemotherapy, the tumor was located in the upper part of your brain and the tumor cells are favorable, you will have no further treatment and will be carefully watched (Observation).
  • If the tumor has completely gone away after Induction chemotherapy, but the tumor was not located in the upper part of your brain and/or the tumor cells are unfavorable, you will have more treatment. You will be randomized to receive either radiation therapy and more chemotherapy (Maintenance) or to receive radiation therapy and then be carefully watched (Observation).


Induction chemotherapy consists of 2 cycles and lasts for a total of about 7 weeks (or about 49 days). Cycle A lasts 3 weeks (21 days) and Cycle B lasts 4 weeks (28 days). All subjects will receive Induction chemotherapy.

Not all subjects will receive Maintenance chemotherapy. If you do receive Maintenance chemotherapy, it will be given after radiation therapy. Maintenance chemotherapy lasts for a total of about 12 weeks (or 84 days).

Who to contact:

PI: Mark Ranalli, MD

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000