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

Cancer type:

Children's cancers
Neuroblastoma

Status:

Results

Phase:

Phase 2

This trial was for children and young people with neuroblastoma that was not responding to treatment or had come back. The trial team looked at a number of different cancer drugs.

The trial was supported by Cancer Research UK. It was open for people to join between 2013 and 2021. The team published results in 2022 and 2024.

More about this trial

Doctors can use surgery, chemotherapy, radiotherapy and targeted cancer drugs to treat neuroblastoma.  Sometimes neuroblastoma continues to grow despite treatment, or it comes back after treatment.

When this happens, there are treatments available that doctors use. But they wanted to find out if other treatments may be helpful for people in this situation.

In this trial they looked at the following chemotherapy drugs:

  • irinotecan
  • temozolomide
  • topotecan

And at these targeted cancer drugs:

  • bevacizumab (Avastin)
  • dinutuximab beta (Qarziba)

The main aims of this trial were to find out:

  • which combinations of treatment work best for neuroblastoma
  • more about the side effects of the different combinations

Summary of results

Trial design
This trial was for children and young people between 1 and 21 years old. They had already had treatment for neuroblastoma.

A total of 225 people took part in this trial. They were put into one of the following treatment groups at random:

  • temozolomide 
  • temozolomide and bevacizumab
  • temozolomide and irinotecan
  • temozolomide, irinotecan and bevacizumab
  • temozolomide and topotecan
  • temozolomide, topotecan and bevacizumab 
  • temozolomide and dinutuximab beta
  • temozolomide, topotecan and dinutuximab beta

These groups weren’t all open to begin with, or at the same time. The groups available changed depending on when people joined the trial.

The research team compared the results for:

  • those who had bevacizumab with those who didn’t
  • those who had irinotecan with those who didn’t
  • those who had topotecan with those who didn't
  • those who had dinutuximab beta and those who didn’t

How well treatment worked
The research team looked at how many people’s cancer got a bit smaller or went away. They found it was:

  • 21 out of 80 people (26%) who had bevacizumab
  • 14 out of 80 people (18%) who didn’t have bevacizumab
  • 12 out of 60 people (20%) who had irinotecan
  • 13 out of 61 people (21%) who didn’t have irinotecan
  • 8 out of 30 people (27%) who had topotecan
  • 7 out of 31 people (23%) who didn’t have topotecan
  • 15 out of 43 people (35%) who had dinutuximab beta
  • 4 out of 22 people (18%) who didn’t have dinutuximab beta

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

  • nearly 7 out of every 10 people (69%) who had bevacizumab
  • just under 6 out of every 10 people (58%) who didn’t have bevacizumab
  • 7 out of every 10 people (70%) who had irinotecan
  • just under 6 out of every 10 people (58%) who didn’t have irinotecan
  • 6 out of every 10 people (60%) who had topotecan
  • just over 5 out of every 10 people (52%) who didn’t have topotecan

The trial team plan to publish the full results for dinutuximab beta separately. We hope to update this summary once those results are available.

When they looked in more detail, the results suggested that those who had both bevacizumab and irinotecan did best. But it is hard to say for sure.

Side effects
Most people taking part had at least 1 side effect. Many were mild or didn’t last long. But some people had more serious side effects.

The team found that:

  • more people who had bevacizumab had side effects than those who didn’t have bevacizumab
  • the most common side effects of bevacizumab were diarrhoea and a drop in blood cells causing increased risk of infection, bruising, bleeding and shortness of breath 
  • more people who had irinotecan and topotecan had side effects than those who didn’t
  • the most common side effect of irinotecan was diarrhoea
  • the most common side effect of topotecan was a drop in blood cells
  • more people who had bevacizumab had protein in their urine
  • more people who had dinutuximab beta had more nerve problems than those who didn’t – these included nerve pain, headaches and tingling sensations
  • fewer people who had dinutuximab beta and chemotherapy had side effects that were a bit worse, compared to those who had chemotherapy alone 

We have more information about the side effects of these treatments in our cancer drugs section:

Conclusion
The trial team concluded that bevacizumab and chemotherapy works better than chemotherapy alone. This is in people with neuroblastoma that has not responded to treatment or has come back. 

They think that irinotecan and bevacizumab together may be better than either treatment on their own. But it’s difficult to say for sure based on the results of this trial. 

They also concluded that having dinutuximab beta and temozolomide could be a useful treatment for this group of people.

They suggest more work is done to find out more about how well different combinations of treatment work. They hope to look at bevacizumab and dinutuximab beta treatment in a new trial called BEACON2.

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

Please note, the articles we link to here are not in plain English. They have been written for healthcare professionals and researchers.

BEACON-Immuno: Results of the dinutuximab beta (dB) randomization of the BEACON-Neuroblastoma phase 2 trial—A European Innovative Therapies for Children with Cancer (ITCC–International Society of Paediatric Oncology Europe Neuroblastoma Group (SIOPEN) trial.
J Gray and others
ASCO meeting, 2022.

Bevacizumab, Irinotecan, or Topotecan Added to Temozolomide for Children With Relapsed and Refractory Neuroblastoma: Results of the ITCC-SIOPEN BEACON-Neuroblastoma Trial
L Moreno and others
Journal of Clinical Oncology. Published online, January 2024 (JCO.23.00458). 

Where this information comes from    
We have based this summary on the information in the articles above. These have been reviewed by independent specialists (peer reviewed Open a glossary item) and published in medical journals. We have not analysed the data ourselves. As far as we are aware, the links we list above are active and the articles are 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 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.

If you have questions about the trial please contact our cancer 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.

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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