An international clinical program for the diagnosis and treatment of children, adolescents and young adults with ependymoma

Trial Overview and Summary

The ependymoma program is a comprehensive program to improve the accuracy of the primary diagnosis and explore different therapeutic strategies in children, adolescents and young adults accordingly. This program is opened to all patients diagnosed with ependymoma below the age of 22 years.

It will include a centralized review of pre and post-operative imaging to assess the completeness of the resection.

It will also include a central review of pathology to confirm the histological diagnosis and to prospectively assess biological markers and molecular subgroups for prospective evaluation of disease subgroups. Further biological evaluations will be coordinated within the linked BIOMECA study.

After surgery and central review of imaging and pathology, patients will be offered the opportunity to undergo second look surgery if possible. Patients will be enrolled in one of 3 different strata according to the outcome of the initial surgical resection (residual disease vs no residual disease), their age or eligibility/suitability to receive radiotherapy.

  • Stratum 1 is a randomised phase III study for patients who have had a complete resection, with no measurable residual disease (as confirmed by centrally reviewed MRI) and are >12 months and <22 years at diagnosis. Those patients will be randomised to receive conformal radiotherapy followed by either 16 weeks of chemotherapy with VEC-CDDP, or observation.
  • Stratum 2 is a randomised phase II study for patients who have inoperable measurable residual disease and who are >12 months and <22 years at diagnosis. Those patients will be randomised to two different treatment schedules of chemotherapy either with VEC or VEC+High Dose Methotrexate. After completion of the frontline chemotherapy, patients will be assessed for response (MRI) and will receive second look surgery when feasible. For those patients who remain unresectable with residual disease despite frontline chemotherapy and for whom second line surgery is not feasible, there will be a study of the safety of a radiotherapy boost of 8Gy that will be administered to the residual tumour immediately after the completion of the conformal radiotherapy. Patients without evidence of residual disease after chemotherapy and/or second look surgery are not eligible for radiotherapy boost. All patients who have not shown progression under chemotherapy will receive as maintenance therapy a 16 week course of VEC –CDDP following completion of radiotherapy.
  • Stratum 3 is a randomized phase II chemotherapy study in children <12 months or those not eligible to receive radiotherapy (see national criteria). These patients will be randomised to receive a dose dense chemotherapy alternating myelosuppressive and relatively non-myelosuppressive drugs at 2 weekly intervals with or without the addition of the histone deacetylase inhibitor, valproate.
  • Registry: Patients that do not fulfil the inclusion criteria of one of the interventional strata will be enrolled and followed up into an observational study which will be analysed descriptively.

Please note that the trials team cannot give individuals clinical advice. Patients and their families should contact their treating physician to discuss trials for which they may be eligible

Chief Investigator: Professor Richard Grundy
UK Coordinating Sponsor: University of Birmingham
Funders: CRUK
Disease Site: Brain
Trial Type: Clinical Trial of an Investigational Medical Product
Status: Open to recruitment
EudraCT No.:
Open to new sites? No
Recruitment start date: February 2016
Anticipated Recruitment end date March 2021
CRCTU Trial Management Team: Children's Cancer Trials Team
Trial E-mail Address: ependymoma@trials.bham.ac.uk

Trial Protocol

Please Note:

Clinical trial protocols are complex technical documents which should only be used for the treatment of subjects taking part in the trial. Patients who are interested in taking part in the trial are advised to talk to their health care professional or refer to CancerHelp website see the link below.

Investigators please ensure you have R&D approval for this specific version of the protocol before using as a reference. 

SIOP Ependymoma II Protocol v3.0 2017-01-17 [.pdf]


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