Trial Information

CCLG Observational Study of the Outcome of Ependymoma in Infants Diagnosed Before Their Third Birthday

OBJECTIVES:

Primary

- To determine the overall survival and event-free survival of all infants diagnosed with
ependymoma before their third birthday.

- To determine the overall survival and event-free survival of infants diagnosed with
ependymoma before their third birthday when treated with standard chemotherapy
comprising vincristine, carboplatin, high-dose methotrexate, cyclophosphamide, and
cisplatin.

Secondary

- To investigate the reasons why the primary tumor was completely resected in patients
who were able to undergo complete resection of the tumor.

- To continue to investigate the biological characteristics of ependymoma.

- To correlate functional imaging studies of ependymoma with biological characteristics
of the tumor.

- To provide a standard treatment regimen for patients with residual disease after
optimal surgery who have already participated in a phase II study.

- To prospectively document renal function, hearing, and neurocognitive late effects
after completion of study treatment.

OUTLINE: This is a multicenter study. Patients are stratified according to extent of prior
surgical resection and presence of metastatic disease (complete resection of tumor vs
metastatic disease at diagnosis vs no complete resection of tumor).

Patients receive vincristine IV on days 1, 15, and 29, carboplatin IV over 1 hour on day 1,
high-dose methotrexate* IV over 24 hours on day 15, cyclophosphamide IV over 1 hour on day
29, and cisplatin IV over 48 hours on days 43 and 44. Treatment repeats every 8 weeks for 7
courses in the absence of disease progression or unacceptable toxicity. Patients with
residual disease after completion of treatment may receive other treatment at the discretion
of the investigator.

NOTE: *Patients initially treated on clinical trial CCLG-CNS-2005-03 who have no residual
disease do not receive high-dose methotrexate in courses 5-7.

Patients undergo observational assessments comprising physical and neurological examination;
MRI/ MRS scanning of the head and spine; and audiology, renal, endocrine, neurocognitive,
and quality of life evaluations periodically for at least 5 years after the completion of
study treatment.