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Observing Young Patients With Ependymoma Undergoing Standard Combination Chemotherapy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00683319
Recruitment Status : Active, not recruiting
First Posted : May 23, 2008
Last Update Posted : September 20, 2013
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Gathering information about how young patients with ependymoma respond to standard combination chemotherapy and learning about the long-term effects of this treatment may help doctors plan better treatment.

PURPOSE: This phase III trial is observing young patients with ependymoma undergoing standard combination chemotherapy.

Condition or disease Intervention/treatment
Brain and Central Nervous System Tumors Cognitive/Functional Effects Long-term Effects Secondary to Cancer Therapy in Children Ototoxicity Drug: carboplatin Drug: cisplatin Drug: cyclophosphamide Drug: methotrexate Drug: vincristine sulfate Procedure: adjuvant therapy Procedure: cognitive assessment Procedure: magnetic resonance imaging Procedure: magnetic resonance spectroscopic imaging Procedure: quality-of-life assessment

Detailed Description:



  • 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.


  • 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.

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Study Type : Observational
Estimated Enrollment : 50 participants
Official Title: CCLG Observational Study of the Outcome of Ependymoma in Infants Diagnosed Before Their Third Birthday
Study Start Date : April 2008
Estimated Primary Completion Date : March 2034

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Overall survival
  2. Event-free survival
  3. Response to chemotherapy, if there is residual disease
  4. Requirement for radiotherapy (i.e., residual disease at the completion of chemotherapy, progressive disease during chemotherapy, or recurrent disease during or after completion chemotherapy)
  5. Late effects of treatment (i.e., ototoxicity and nephrotoxicity at the completion of chemotherapy and neurocognitive outcomes at 5, 7, 11, and 16 years of age)

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 2 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed WHO grade 3 anaplastic (malignant) ependymoma or WHO grade 2 ependymoma, including the following variants:

    • Papillary
    • Cellular
    • Clear cell
    • Tanycytic
  • No myxopapillary ependymoma, subependymoma, or ependymoblastoma
  • Meets 1 of the following criteria:

    • Has undergone complete resection of the primary tumor (prior to starting chemotherapy)

      • Two or more surgical procedures to achieve complete resection allowed
    • Metastatic disease at diagnosis (with or without complete resection of the primary tumor)
    • Unable to undergo complete resection of the primary tumor (with or without metastatic disease)
  • Patients with measurable residual disease (primary or metastatic disease) are eligible provided they undergo treatment on clinical trial CCLG-CNS-2005-03 prior to entering this study
  • Has undergone surgical resection OR completed treatment on clinical trial CCLG-CNS-2005-03 within the past 3 weeks
  • Patients who are unable to tolerate chemotherapy or who do not receive treatment according to the CCLG guidelines for ependymoma due to parental preference or recommendation from the treating physician are eligible


  • Able to tolerate IV hydration
  • No active infection


  • See Disease Characteristics

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00683319

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United Kingdom
Birmingham Children's Hospital
Birmingham, England, United Kingdom, B4 6NH
Addenbrooke's Hospital
Cambridge, England, United Kingdom, CB2 2QQ
Leeds Cancer Centre at St. James's University Hospital
Leeds, England, United Kingdom, LS9 7TF
Royal Liverpool Children's Hospital, Alder Hey
Liverpool, England, United Kingdom, L12 2AP
Great Ormond Street Hospital for Children
London, England, United Kingdom, WC1N 3JH
Royal Manchester Children's Hospital
Manchester, England, United Kingdom, M27 4HA
Queen's Medical Centre
Nottingham, England, United Kingdom, NG7 2UH
Children's Hospital - Sheffield
Sheffield, England, United Kingdom, S10 2TH
Aberdeen Royal Infirmary
Aberdeen, Scotland, United Kingdom, AB25 2ZN
Royal Hospital for Sick Children
Glasgow, Scotland, United Kingdom, G3 8SJ
Sponsors and Collaborators
Children's Cancer and Leukaemia Group
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Principal Investigator: Martin W. English, MD Birmingham Children’s Hospital

Layout table for additonal information Identifier: NCT00683319     History of Changes
Other Study ID Numbers: CDR0000590666
First Posted: May 23, 2008    Key Record Dates
Last Update Posted: September 20, 2013
Last Verified: June 2009
Keywords provided by National Cancer Institute (NCI):
long-term effects secondary to cancer therapy in children
cognitive/functional effects
childhood infratentorial ependymoma
childhood supratentorial ependymoma
Additional relevant MeSH terms:
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Nervous System Neoplasms
Central Nervous System Neoplasms
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms by Site
Nervous System Diseases
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Antimetabolites, Antineoplastic