At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- 16 weeks of VEC + CDDP (drug), VEC + HD-MTX (drug), Chemotherapy + Valproate (drug), Conformal radiotherapy (radiation)
- How long the study runs
- Study runs about 194 months (dates as stated)
- About the drug or intervention
- 16 weeks of VEC + CDDP — drug: Days 1-36-71-106: Vincristine: 1.5 mg/m² (maximal dose 2 mg) i.v.; Days 1-3-36-38-71-73-106-108: Etoposide: 100 mg/m² infused over 60 minutes; Days 1-36-71-106: Cyclophosphamide: 3000 mg/m² in 3 divided infusions (1000 mg/m²/infusion) infused over 60 minutes; Days 22-57-92: Cisplatin: 80 mg/m² over 4 hours + Vincristine:1.5 mg/m² (maximal dose 2 mg) i.v. · VEC + HD-MTX — drug: Days 1-22-43: Vincristine: 1.5 mg/m² (maximal dose 2 mg) i.v.; Days 1-3-22-24-43-45: Etoposide: 100 mg/m² infused over 60 minutes; Days 1-22-43: Cyclophosphamide: 3000 mg/m² in 3 divided infusions (1000 mg/m²/infusion) infused over 60 minutes; Days 15-36-57: Administer methotrexate at 8000 mg/m² as a 24 hour IV infusion on days 15-36-57. · Chemotherapy + Valproate — drug: Days 1-57-113-169-225-281-337: Vincristine and Carboplatin; Days 15-71-127-183-239-295-351: Vincristine and Methotrexate; Days 29-85-141-197-253-309-365: Vincristine and Cyclophosphamide; Days 43-44-99-100-154-155-211-212-267-268-323-324-379-380: Cisplatin 2-day continuous infusion. · Conformal radiotherapy — radiation: Conformal radiotherapy: 59.4Gy (children \<18 months or with risk factors: 54Gy). · VEC — drug: D1: Vincristine: 1.5 mg/m² (maximal dose 2 mg) i.v.; D1-D3: Etoposide: 100 mg/m² infused over 60 minutes; D1: Cyclophosphamide: 3000 mg/m² in 3 divided infusions (1000 mg/m²/infusion) infused over 60 minutes; D22: Vincristine: 1.5 mg/m² (maximal dose 2 mg) i.v.; D22-D24: Etoposide: 100 mg/m² infused over 60 minutes; D22: Cyclophosphamide: 3000 mg/m² in 3 divided infusions (1000 mg/m²/infusion) infused over 60 minutes; D43: Vincristine: 1.5 mg/m² (maximal dose 2 mg) i.v.; D43-D45: Etoposide: 100 mg/m² infused over 60 minutes; D43: Cyclophosphamide: 3000 mg/m² in 3 divided infusions (1000 mg/m²/infusion) infused over 60 minutes · Chemotherapy — drug: Days 1-57-113-169-225-281-337: Vincristine and Carboplatin; Days 15-71-127-183-239-295-351: Vincristine and Methotrexate; Days 29-85-141-197-253-309-365: Vincristine and Cyclophosphamide; Days 43-44-99-100-154-155-211-212-267-268-323-324-379-380: Cisplatin 2-day continuous infusion. · conformal radiotherapy +/- boost — radiation: Conformal radiotherapy: 59.4Gy (children \<18 months or with risk factors: 54Gy).
- Patient visit burden
- Not specified by the sponsor
In plain English
This international study looks at better ways to diagnose and treat children and young people with a type of brain tumour called childhood ependymoma. After initial surgery, patients are placed into one of three treatment groups (called strata) based on things like their age, whether any tumour remains after surgery, and whether they can have radiotherapy. Those who do not fit any treatment group are still followed up in an observational part of the study.
Who can take part
- Under 22 years old at diagnosis
- Newly diagnosed brain (intracranial) ependymoma of WHO grade II or III, confirmed by expert review
- Groups 1 and 2: over 12 months old, no spread of tumour to the spine, no previous radiotherapy or chemotherapy (except steroids), fit for radiotherapy and chemotherapy, and healthy enough bone marrow, liver and kidneys. Group 1 has no tumour left after surgery; Group 2 has tumour left that cannot be operated on
- Group 3: children under 12 months old, or those who cannot have radiotherapy because of age, where the tumour is, or parent/doctor choice
- Not pregnant or breastfeeding, with a negative pregnancy test if of childbearing age, and using effective contraception during treatment and for 6 months after
- Able and willing to attend scheduled visits, treatment and tests, with no other illness or infection that would make chemotherapy unsafe
Who may not be able to
- A tumour other than a primary brain ependymoma, or a diagnosis made before this trial opened
- WHO grade I ependymoma (including myxopapillary ependymoma and subependymoma) or a tumour in the spinal cord
- Taking part in another ependymoma treatment trial, or having other anti-tumour treatment at the same time
- Unable to tolerate chemotherapy or intravenous fluids, or having certain existing gut or fluid problems (such as mouth ulcers, stomach ulcers, inflammatory bowel disease, ascites or fluid on the lungs)
- Groups 1 and 2: unable to have radiotherapy, or MRI scan results that cannot be confirmed
- Group 3: existing severe liver or kidney damage, family history of severe epilepsy, an undiagnosed mitochondrial disorder found by trial screening, or high blood ammonium and lactate levels (1.5 times the normal upper limit or more)
What taking part involves
- • Surgery first, then placement into one of three treatment groups
- • Treatment may include radiotherapy and/or chemotherapy with study drugs, depending on the group
- • Patients not fitting a treatment group are followed up in an observational study instead
- • MRI scans and tests on spinal fluid to check the tumour
Time commitment: Not stated exactly how many visits or how long the trial lasts — patients and parents must be able to attend scheduled visits, treatment, blood tests and other study procedures — ask the trial team.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- Up to 22 Years
- Who
- All
- Number of participants
- 536
- Started
- 2015-06-02
- Last checked
- 2024-04
Plain English Summary
What is this study?
- • Testing a new treatment for childhood ependymoma
- • Phase2/Phase3 - 536 participants
- • The overall aim of this project is to improve the outcome of patients diagnosed with ependymoma by improving and harmonising the staging and the standard of care of this patient population and to improve the investigators understanding of the underlying biology thereby informing future treatment
Who can take part?
- • Ages Up to 22 Years
- • Diagnosed with childhood ependymoma
Where?
- • Nottingham - Queen's Medical Centre
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The overall aim of this project is to improve the outcome of patients diagnosed with ependymoma by improving and harmonising the staging and the standard of care of this patient population and to improve the investigators understanding of the underlying biology thereby informing future treatment. The program will evaluate new strategies for diagnosis (centralized reviews of pathology and imaging) and new therapeutic strategies in order to develop treatment recommendations. Patients will be stratified into different treatment subgroups according to their age, the tumour location and the outcome of the initial surgery. Each subgroup will be studied in a specific randomised study to evaluate the proposed therapeutic strategies. Stratum 1: The aim of the stratum 1 is to evaluate the clinical impact of 16-week chemotherapy regimen with VEC-CDDP following surgical resection and conformal radiotherapy in terms of progression free survival in patients who are \> 12 months and \< 22 years at diagnosis, with completely removed intra cranial Ependymoma. Stratum 2: This stratum is designed as a phase II trial for patients who are \> 12 months and \< 22 years at diagnosis, with residual disease to investigate the possible activity of HD-MTX by giving to all patients the benefit of VEC chemotherapy whilst randomising half of patients to receive additional HD-MTX. Patients will receive conformal radiotherapy (cRT). For patients who remain with a residual inoperable disease after induction chemotherapy and cRT, an 8 Gy boost of radiotherapy to the residual tumour will be delivered immediately after the end of the cRT. Stratum 3 This stratum is designed as a phase II trial to evaluate the benefit of postoperative dose intense chemotherapy administered alone or in combination with valproate in children \<12 months of age or those not eligible to receive radiotherapy .
More detail
The Ependymoma Program is a comprehensive program to improve the accuracy of the primary diagnosis of ependymoma 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 centralised 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. The biological markers 1q and 6q gain, Tenascin C status, NELL2 and LAMA2, RELA-fusion and molecular subgroup by methylation array will be prospectively assessed 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. These 3 different strata correspond to 3 therapeutic strategies according to the patient status. 1. Stratum 1 is designed as 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. 2. Stratum 2 is designed as 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 (VEC +HD-MTX). 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 8 Gy that will be administered to the residual tumour immediately after the completion of the conformal radiotherapy. Patients without evidence of residual disease after the chemotherapy and/or a 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. 3. Stratum 3 is designed as a randomised phase II chemotherapy study in children \<12 months of age or those not eligible to receive radiotherapy. 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 via an observational study which will be analysed descriptively.
How this trial compares with your answers
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What we know so far
Still need:
- • Tell us your age for better matching
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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Up to 22 Years
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Patients with centrally and confirmed by a biopsy intracranial ependymoma meeting the following criteria will be...
- ✓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...
- ✓Males and females of reproductive age and childbearing potential with effective contraception for the duration of...
See the full criteria
Where Is This Study? (1 UK site)
Queen's Medical Centre
Nottingham, United Kingdom
How to Get in Touch
Pierre LEBLOND, MD
Sponsor contactCONTACT