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

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Brain (and spinal cord) tumours

Children's cancers

Status:

Closed

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 30 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 had another type of cancer in the past 5 years
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 )
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 common side effects of erlotinib are:

Skin problems such as acne, or a dry, red rash
diarrhoea
feeling or being sick
sore mouth
chills
fever
loss of appetite
increased risk of infection
nose bleeds
a hole or tear in the lining of the gut
tiredness (fatigue)
changes to your nails
hair thinning
eye problems such as sore, red eyes
changes to the way your liver and kidneys work
breathing problems such as breathlessness and a cough
tummy (abdomen) pain

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, liver and heart works
slow healing of wounds
breathing problems
difficulty swallowing
indigestion
feeling very thirsty (dehydratiom)
loss of appetite
feeling sick
diarrhoea
tummy (abdomen) pain
joint pain

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