A trial looking at etoposide for children and young people with ependymoma (CNS 2001 04)

Cancer type:

Brain (and spinal cord) tumours
Children's cancers




Phase 2

This trial was for children and young people with ependymoma that had not been completely removed or had come back after treatment.

The trial was open for people to join between 2002 and 2010. The team published the results in 2022.

More about this trial

Ependymoma is a type of brain tumour. Doctors usually treat ependymoma with surgery and radiotherapy. But it is not always possible to completely remove the tumour. And it can come back after treatment. If this happens, it is more difficult to treat.

Doctors wanted to find out if a chemotherapy drug called etoposide may be useful for children and young people in this situation. When this trial was done, etoposide was already being used to treat other types of cancer.

The main aim of this trial was to find out how well etoposide works for children and young people with ependymoma that has not been removed or has come back.

Summary of results

Trial design
This trial was for children and young people up to 21 years old who had ependymoma. It was for people who’d had treatment, but their brain tumour had not gone away or it had come back. Doctors were not able to remove it with surgery.

The people taking part had etoposide through a drip into a vein. They had treatment 3 days a week for 3 weeks. And then a week with no treatment. Each 4 week period is called a cycle of treatment. They had up to 6 cycles.

The research teams looked at scans before, during and after treatment to see how well it was working.

A total of 25 people joined this trial. They were between 2 and 17 years old. Three people didn’t have treatment as part of the trial. The other 22 people had between 1 and 6 cycles of treatment.

The trial team looked at how well the treatment worked. They were able to assess this in 21 people (96%) who took part. They found that the brain tumour had:

  • gone away in 2 people (9%)
  • got smaller in 7 people (32%)
  • stayed the same in 5 people (23%)
  • continued to grow in 7 people (32%)

They looked at how many people’s cancer had not started to grow again within 1 year and 3 years of joining the trial. They found it was:

  • 5 people (23%) at 1 year
  • 3 people (14%) at 3 years

They also looked at how many people were living 1 year and 5 years after joining the trial. They found it was:

  • 13 people (59%) at 1 year
  • 4 people (18%) at 5 years

These results are similar to trials that looked at having etoposide tablets. In this trial people had etoposide into a vein (intravenous treatment).

Side effects
Everyone who had treatment had at least 1 side effect. Some were mild or didn’t last long. But most people had at least 1 that was more severe.

The most common of the more severe side effects was a drop in blood cells. This includes a drop in white blood cells, red blood cells and clotting cells (platelets).

We have more information about the side effects of etoposide in our Cancer drugs section.

The trial team concluded that etoposide may be useful for some children and young people with ependymoma that has come back and can’t be removed with surgery. But it doesn’t work for everyone. They say it might be helpful to use etoposide in combination with other treatments.

They also suggest more work is done looking at biomarkers  Open a glossary itemto help find out who is likely to benefit from treatment.

More detailed information
There is more information about this research in the reference below. 

Please note, this article is not in plain English. It has been written for health care professionals and researchers.

Journal articles
Phase II study of intravenous etoposide in patients with relapsed ependymoma (CNS 2001 04) 
John R Apps and others
Neuro-Oncology Advances, 2022. Volume 4, Issue 1

Where this information comes from    
We have based this summary on the information in the article above. This has been reviewed by independent specialists (peer reviewed Open a glossary item) and published in a medical journal. We have not analysed the data ourselves. As far as we are aware, the link we list above is active and the article is free and available to view.

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Sue Picton
Dr Linda Lashford

Supported by

Cancer Research UK Children's Cancer Trials Team
Children’s Cancer and Leukaemia Group (CCLG)
University of Birmingham
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer

If you have questions about the trial please contact our cancer information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle 247

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Wendy took part in a new trial studying the possible side effect of hearing loss

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"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”

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