A trial looking at different types of treatment for children and young people with neuroblastoma (BEACON - Neuroblastoma)

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Cancer type:

Children's cancers
Neuroblastoma

Status:

Open

Phase:

Phase 2

This study is for children and young people with neuroblastoma that is not responding to treatment or has come back. 

It includes the following trial treatments: 

  • Bevacizumab (this arm is now closed)
  • Cyclophosphamide
  • Dinutuximab beta
  • Irinotecan (this arm is now closed)
  • Temozolomide
  • Topotecan

Cancer Research UK supports this trial.

More about this trial

This trial is for children and young people up to and including the age of 21. We use the term 'you' in this summary, but if you are a parent, we are referring to your child.

Doctors can use different treatments for neuroblastoma including surgery, chemotherapy and radiotherapy.

Sometimes neuroblastoma gets worse despite having treatment and, even if it responds well to treatment to begin with, it may come back later on. 

In this situation, there are different treatments that doctors can use. Irinotecan is a type of chemotherapy drug doctors might use. Recruitment into the irinotecan treatment arm is now closed as enough people have already joined this group.  

Researchers wanted to find out if having a drug called bevacizumab (Avastin) could help children with neuroblastoma. Bevacizumab is a type of targeted drug called a monoclonal antibody. It works by targeting a protein called vascular endothelial factor that helps cancers to grow blood vessels.
 
Doctors already use bevacizumab to treat a number of adult cancers. It is now being looked at in trials to see if it also works in children. Recruitment into the bevacizumab treatment arm is also closed as enough people have already joined this group. 

The most recent drug added to the trial is called dinutuximab beta (Qarziba). Dinutuximab beta is a targeted cancer drug called a monoclonal antibody. It targets a protein called GD2 that is sometimes produced in excess by neuroblastoma cells.
 
In this trial, you might have dinutuximab beta in combination with temozolomide and topotecan. Dinutuximab beta is already a treatment for neuroblastoma that is at a high risk of coming back after treatment. But this is the first time that this combination will be tested in people with neuroblastoma in Europe. 

You may also have a drug called cyclophosphamide, which is a commonly used chemotherapy to treat cancer.

The aims of this trial are to:

  • Find out whether having irinotecan or topotecan as well as temozolomide helps children and young people with neuroblastoma that isn’t responding to other treatment or has come back afterwards
  • See if adding bevacizumab or dinutuximab beta to chemotherapy helps children and young people 
  • Learn more about the side effects of the different drug combinations

Who can enter

You may be able to enter this trial if you

  • Have neuroblastoma that has come back or neuroblastoma that isn’t responding to treatment – your doctor can explain this to you
  • Have neuroblastoma that doctors can see and measure on a scan
  • Are at least 1 year old but have not yet reached your 22nd birthday
  • Have satisfactory blood test results
  • Are willing to use reliable contraception during the trial and for 6 months afterwards if you are sexually active and there is any chance you or your partner could become pregnant

You cannot enter this trial if you

  • Have neuroblastoma that has spread to your brain and is causing bleeding – you can take part if it has spread to your brain as long as it isn’t bleeding
  • Have already had temozolomide
  • Have had major surgery in the last 2 weeks
  • Have had radiotherapy or chemotherapy in the last 2 weeks
  • Have had radiotherapy to your brain or spine, or targeted radiotherapy called MIBG in the last 6 weeks
  • Have had a stem cell transplant using your own cells in the last 8 weeks or a transplant using donor cells in the last 3 months
  • Have had an experimental drug in the last 2 weeks
  • Have had treatment with dinutuximab beta in combination with another chemotherapy
  • Have had a biopsy  Open a glossary itemin the last 2 days
  • Are known to be very sensitive to anything in the trial drugs or to drugs made in a similar way
  • Have neurological problems, problems with nerves in your extremities such as hands, feet or arms, or seizures (fits) – the trial team can advise you about this
  • Have an infection that isn’t controlled
  • Are planning to have a live vaccine
  • Have any other medical condition that the trial team think could make it unsafe for you to take part

Trial design

This is a phase 2 trial. The doctors need around 224 children and young people to take part.

It is a randomised trial. This trial is in two parts:

  • Bevacizumab randomisation
  • Dinutuximab beta randomisation 

Bevacizumab randomisation 
The first 160 children and young people who took part were put into one of the following treatment groups by a computer:

  • Temozolomide alone - this group is now closed
  • Bevacizumab and temozolomide - this group is now closed
  • Irinotecan and temozolomide – this group is now closed 
  • Bevacizumab, irinotecan and temozolomide – this group is now closed 
  • Temozolomide and topotecan – this group is open 
  • Bevacizumab, topotecan and temozolomide - this group is now closed

Neither the patient nor their doctor were able to decide which group they were in.

Dinutuximab beta randomisation (open to recruitment) 
The doctors need around 64 children and young adults to take part in this part of the trial. 

The children and young people taking part will be put into one of the following groups by computer: 

  • Temozolomide and topotecan 
  • Dinutuximab beta, temozolomide and topotecan 

Neither you nor your doctor will be able to decide which group you are in. 

You have treatment in cycles of treatment that last 4 weeks. You can have up to 6 cycles of treatment in total, taking around 24 weeks.

If you are put into the temozolomide and topotecan group, and your cancer does not get better or worse within 30 months, you may be allowed to have treatment with dinutuximab beta, topotecan and cyclophosphamide. This is called crossover.
 
Dinutuximab beta, topotecan and cyclophosphamide are drugs that you have through a drip into a vein. Dinutuximab beta is given continuously over 7 days. Temozolomide is a tablet that you swallow whole. 

Blood, saliva and tumour samples
During the trial, the team will take some extra blood samples to look for biomarkers. These are substances that doctors can measure to help them tell how a disease is developing or how a treatment is working. 

They will also get a sample of your tumour that was removed when you had surgery or a biopsy in the past. If you have any more surgery as part of your neuroblastoma treatment, they will ask for another sample of the tumour.

Before you start treatment, they will ask you to give an optional sample of saliva or blood that they can use to study your DNA.

Hospital visits

You see the trial team and have some tests before you start treatment. The tests include

During treatment, you see the trial team and have regular blood tests. You have a scan after every 2 cycles of treatment. You may have more bone marrow tests if you had neuroblastoma cells in your bone marrow at the beginning of the trial.

When you finish treatment, you see the trial team again. You have a physical examination, blood tests, urine tests, and another X-ray of your hand and wrist. You also have a CT or MRI scan and a PET-CT or MIBG scan. You may need to have another bone marrow test.

Side effects

As only a small number of children have had bevacizumab so there may be side effects we don’t know about yet. In adults, the most common side effects include

Doctors don’t know if bevacizumab affects bone growth. The trial team monitored the growth of the children taking part and took X-rays of their hand and wrist to check this.

The most common side effects of irinotecan include

  • Diarrhoea which can be severe
  • Changes to the way your liver works
  • Temporary hair loss
  • A drop in blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness

The most common side effects of topotecan include

  • Feeling or being sick
  • A drop in blood cells
  • Tummy (abdominal) pain
  • High temperature (fever)
  • Poor appetite
  • Hair loss
  • Itchy skin
  • Tiredness

The possible side effects of temozolomide include

  • Feeling or being sick
  • A drop in blood cells
  • Swelling of your gums
  • Weight loss
  • Hair loss
  • Symptoms of brain inflammation (this is uncommon)
  • A severe infection called sepsis (this is uncommon)

The possible side effects of dinutuximab beta include:

  • Pain in different parts of your body that can be severe
  • High temperature (fever)
  • Cough 
  • Severe allergic reactions
  • Eye changes which are usually temporary
  • A build-up of fluid in your body which can cause low blood pressure and difficulty breathing 
  • Feeling or being sick
  • Diarrhoea or constipation
  • A drop in the number of blood cells which can cause infection and bleeding
  • Nerve changes which can be permanent 

Cyclophosphamide is a commonly used drug used to treat cancer and has similar side-effects to temozolomide and topotecan chemotherapy.

We have more information about the side effects of:

Location

Aberdeen
Belfast
Birmingham
Bristol
Cambridge
Glasgow
Leeds
Liverpool
London
Manchester
Newcastle upon Tyne
Sheffield
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 Lucas Moreno

Supported by

Cancer Research UK
Experimental Cancer Medicine Centre (ECMC)
Eusa Pharma
Imagine for Margo
NIHR Clinical Research Network: Cancer
Roche
Solving Kids Cancer
University of Birmingham

Other information

This is Cancer Research UK trial number CRUK/11/056.

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

9185

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

Over 60,000 cancer patients enrolled on clinical trials in the UK last year.

Last reviewed:

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