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Improving diagnosis and treatment for children with ependymoma

An International Clinical Program for the Diagnosis and Treatment of Children, Adolescents and Young Adults With Ependymoma

Phase2; Phase3 Interventional Centre Leon Berard · NCT02265770

This study is trying to improve how children with ependymoma are diagnosed and treated by organizing their care and testing new treatment strategies based on their specific needs.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment536 (estimated)
AgesN/A to 22 Years
SexAll
SponsorCentre Leon Berard Academic / other
Drugs / interventionschemotherapy, methotrexate
Locations40 sites (Graz and 39 other locations)
Trial IDNCT02265770 on ClinicalTrials.gov

What this trial studies

This program aims to enhance the outcomes for children diagnosed with ependymoma by standardizing staging and care while deepening the understanding of the disease's biology. It involves centralized reviews of pathology and imaging to ensure accurate diagnosis and treatment recommendations. Patients under 22 years old will be stratified into different treatment groups based on age, tumor location, and surgical outcomes, with each group undergoing specific randomized studies to evaluate new therapeutic strategies. The program includes a comprehensive assessment of biological markers to inform future treatments.

Who should consider this trial

Good fit: Ideal candidates are children and young adults under 22 years old with newly diagnosed intracranial ependymoma confirmed by central pathology.

Not a fit: Patients with ependymoma who are over 22 years old or those with contraindications to the proposed treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this program could significantly improve survival rates and quality of life for children with ependymoma.

How similar studies have performed: Other studies have shown promise in improving outcomes for pediatric ependymoma, but this program's comprehensive approach is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
After Initial surgery, patients will be enrolled in one of 3 different interventional strata where they will be offered a set of therapeutic interventions based on the outcome of the intervention (no measurable residue vs residual inoperable disease), their age and/or their eligibility /suitability to receive radiotherapy.

Patients with centrally and histologically confirmed intracranial ependymoma meeting the following criteria will be enrolled into one of interventional stratum:

* Age \< 22 years old at diagnosis
* Newly diagnosed intracranial ependymoma of WHO grade II-III confirmed by central pathological review
* Post-menarchal female not pregnant or nursing (breast feeding) and with a negative beta-HCG pregnancy test prior to commencing the trial
* Males and females of reproductive age and childbearing potential with effective contraception for the duration of their treatment and 6 months after the completion of their treatment
* No contraindication to the use if one of the study drugs proposed by the protocol
* Patients and/or their parents or legal guardians willing and able to comply with scheduled visits, treatment plan, laboratory tests and other study procedure
* No co-existent unrelated disease at the time of study entry that would render the patient unable to receive chemotherapy
* No signs of infection.

Common inclusion criteria for Strata 1 and 2:

* Age \> 12 months and \< 22 years at time of study entry
* No metastasis on spinal MRI and on CSF cytology assessments
* No previous radiotherapy
* No previous chemotherapy (except steroids)
* No medical contraindication to radiotherapy and chemotherapy
* Adequate bone marrow, liver and renal functions

Specific inclusion criteria for Stratum 1:

• No residual measurable ependymoma based on the central neuroradiological review (R0-1-2)

Specific inclusion criteria for Stratum 2:

• Residual non reoperable measurable ependymoma based on the central neuroradiological review (R3-4)

Inclusion criteria for Stratum 3:

* Children younger than 12 months at time of entry to study or any children ineligible to receive radiotherapy due to age at diagnosis, tumour location or clinician / parent decision and according to national criteria
* Adequate bone marrow, liver and renal functions
* No previous chemotherapy and radiotherapy
* No contraindication to chemotherapy Patients that do not fulfill the inclusion criteria of one of the interventional strata will be enrolled and followed up into an observational study and descriptive analysis will be performed.

EXCLUSION CRITERIA for all interventional strata:

* Tumour entity other than primary intracranial ependymoma
* Primary diagnosis predating the opening of SIOP Ependymoma II
* Patients with WHO grade I ependymoma including ependymoma variants: myxopapillary ependymomas and subependymomas,patients with spinal cord location of the primary tumour
* Participation within a different trial for treatment of ependymoma
* Contraindication to one of the IMP used according to the SmPCs
* Concurrent treatment with any anti-tumour agents
* Inability to tolerate chemotherapy
* Unable to tolerate intravenous hydration
* Pre-existing mucositis, peptic ulcer, inflammatory bowel disease ascites, or pleural effusion.

Strata 1 and 2:

* Ineligible to receive radiotherapy
* Patient for whom imaging remains RX despite all effort to clarify the MRI conclusion

Stratum 3:

* Pre-existing severe hepatic and/or renal damage
* Family history of severe epilepsy
* Presence of previously undiagnosed mitochondrial disorder detected by screening as part of trial
* Elevated blood ammonium and lactate level ≥ 1.5 x upper limit of the normal

Where this trial is running

Graz and 39 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Childhood Ependymoma
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.