A trial of targeted cancer therapies for children and young people with a brain tumour (BIOMEDE)

Cancer type:

Brain (and spinal cord) tumours
Children's cancers

Status:

Open

Phase:

Phase 2

This trial is looking at targeted cancer therapies to treat a type of brain tumour called diffuse intrinsic pontine glioma (DIPG).

This trial is for children and young people up to the age of 25. We use the term 'you' in this summary, but of course if you are a parent, we are referring to your child.

Cancer Research UK supports this trial. 

More about this trial

The standard treatment Open a glossary item for diffuse intrinsic pontine glioma is radiotherapy. Unfortunately, for many people, radiotherapy isn’t a cure and the tumour is likely to come back within a year.

The trial team think that giving a targeted cancer drug (a biological therapy Open a glossary item) alongside standard radiotherapy might be better. But they aren’t sure. 

Some targeted cancer drugs work by blocking pathways in cancer cells that tell them to grow and divide. These are called cancer growth blockers. Certain substances (biomarkers) in the tissue of the tumour show that these pathways are active. 

As part of the trial researchers take a small sample of your tumour tissue (biopsy Open a glossary item) to look for these biomarkers. Depending on which biomarkers are present you have one of the following as well as radiotherapy:

The aim of this trial is to find how well erlotinib, everolimus and dasatinib with radiotherapy works for children and young people with diffuse intrinsic pontine glioma.

Who can enter

The following bullet points list the entry conditions for this trial. Talk to your doctor or the trial team if you are unsure about any of these. They will be able to advise you. 

You may be able to join this trial if all of the following apply. You

  • Have diffuse intrinsic pontine glioma
  • Are able to have a sample of tissue (biopsy) of your tumour taken unless a sample is available from a biopsy done when you were diagnosed 
  • Are able to have radiotherapy to the brain
  • Have satisfactory blood test results
  • Need quite a lot of help to care for yourself (Karnofsky performance scale 50 or more) or you get dressed but need to lie down for much of the day and only take part in quiet play and activities (Lansky play scale 50 or more)
  • Are willing to use reliable contraception during treatment and for 6 months afterwards if there is any chance you or your partner could become pregnant
  • Are aged between 6 months old and 25 years old

You cannot join this trial if any of these apply. 

  • You have a type of brain tumour called pilocytic astrocytoma or ganglioma
  • You have had a major bleed within your tumour   
  • You are having other cancer treatments  
  • You have had radiotherapy to the brain stem for another cancer 
  • You have moderate to severe problems affecting other body organs such as the heart, kidneys, liver or lungs
  • You have a medical condition called congenital galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption – your doctor can tell you this
  • You have another medical or mental health condition that the trial team think could affect you taking part
  • You are pregnant or breastfeeding

Trial design

This is a phase 2 trial. The team need 150 children and young people from the UK and Europe to take part.

Everyone has 6 weeks of radiotherapy. Your doctor will talk to you about this. 

The trial team look at a sample of tissue (biopsy) of your tumour for the biomarkers of the pathways. 

If your biopsy shows that you have the biomarkers you will be randomised Open a glossary item to have 1 of 3 treatments.

You are put into treatment groups by a computer. The 3 groups are:

  • erlotinib
  • everolimus
  • dasatinib

Neither you or your doctor can choose which group you are in. 

study diagran

If there isn’t enough cancer tissue from the biopsy to look for the biomarkers you have dasatinib.

It might be that you are not able to have all of the drugs used in the trial. This is because the way they work depends on the biomarkers you do, or don’t have on your tumour. The trial doctors can tell you more about this if it applies to you. 

Erlotinib is a tablet you take once a day. You take it on an empty stomach, at least an hour before food or 2 hours after food.

Everolimus is a tablet you take once a day. 

Dasatinib is a tablet you take twice a day. 

You start taking your tablets the same day you start radiotherapy. You continue treatment for up to 18 months as long as it is helping and the side effects aren’t too bad. 

Samples for research
You might need to have a lumbar puncture Open a glossary item test as part of your normal care. 

If you have the lumbar puncture the trial team might ask your permission to take an extra sample of the fluid surrounding your spinal cord (the CSF). They will also ask for an extra blood sample. The doctors want to see how much of the drug you are having is in the CSF and compare this to how much is in the blood sample. 

You don’t have to agree to give these samples. You can still take part in the trial.

Hospital visits

You see the doctor to have some tests before taking part in this trial. These tests include:

  • a physical examination
  • blood tests
  • a test to see how your brain and nervous system works (a neurological examination)
  • heart trace (ECG Open a glossary item)
  • chest x-ray
  • MRI scan 

You see the doctor every week during the time you have radiotherapy. You then see the doctor every 2 weeks or monthly. You’ll have regular blood tests during the time you have your treatment. Once you have finished you radiotherapy you have a MRI scan. You then have a scan every 2 to 3 months. 

At the end of treatment you see the doctor for:

  • a physical examination
  • blood tests
  • a test to see how your brain and nervous system works (a neurological examination)
  • MRI

You then see them every 3 to 4 months.

Side effects

The most common side effects of erlotinib are:

The most common side effects of everolimus are:

  • a drop in blood cells causing an increased risk of infection, bruising and bleeding
  • general swelling caused by a build up of fluid in the body
  • high blood pressure
  • coughing 
  • nose bleeds
  • increase in the amount of sugar and fats (lipids) in the blood
  • sore, red mouth
  • taste changes
  • rash, itchy or dry skin 
  • headaches
  • tiredness (fatigue)
  • feeling irritable or aggressive
  • difficulty sleeping
  • changes to the way your kidneys and liver works
  • slow healing of wounds
  • breathing problems

The most common side effects of dasatinib are:

  • a drop in blood cells causing an increased risk of infection, bruising and bleeding
  • a high temperature (fever) with a low number of white blood cells (febrile neutropenia)
  • loss of appetite
  • weight loss or weight gain
  • difficulty sleeping
  • headaches
  • drowsiness
  • depression
  • blurred vision
  • ringing in the ears (tinnitus) 
  • heart problems, heart failure and fluid around the heart
  • difficulty breathing 
  • coughing
  • chest pain
  • diarrhoea
  • feeling or being sick
  • tummy (abdominal) pain
  • rash or red, dry, itchy skin
  • sore muscles and joints
  • tiredness (fatigue)
  • general swelling caused by fluid build up in the body

We have more information about:

We have information about the side effects of radiotherapy to the brain.

Your doctor will talk to you about the side effects of all the treatments used before you agree to take part in the trial.

Location

Birmingham
Bristol
Edinburgh
Glasgow
Leeds
Liverpool
London
Manchester
Newcastle upon Tyne
Nottingham
Oxford
Southampton
Sutton

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 Darren Hargrave

Supported by

Cancer Research UK
Institute of Cancer Research (ICR)
The Brain Tumour Charity

Other information

This is Cancer Research UK trial number CRUK/15/006.

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

13384

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

Cara took part in a clinical trial

A picture of Cara

"I am glad that taking part in a trial might help others on their own cancer journey.”

Last reviewed:

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