CompletedPhase IIInterventional

A trial of BEvACizumab added to temozolomide +/- irinOtecan for children with refractory/relapsed Neuroblastoma

Neuroblastoma is a rare type of cancer that develops from nerve cells, mostly affecting babies and young children. While initial treatments can work well, the cancer sometimes comes back or stops responding to therapy. When this happens, doctors need better, more effective treatment choices. This clinical trial, called BEACON, is testing whether adding a drug called bevacizumab to standard chemotherapy works better than chemotherapy alone. Bevacizumab works by blocking the blood supply that tumours need to grow. The trial involves children and young people aged 1 to 21 years across the UK. By comparing different treatment combinations, researchers hope to discover which approach is most effective at shrinking tumours and keeping the disease under control, while carefully monitoring safety and quality of life.

At a glance

Status

Completed

Phase

Phase II

Sponsor

University of Birmingham

Enrolment target

224

Start

01 Apr 2013

Estimated completion

19 Feb 2021

Neuroblastoma in children aged ≥1 to ≤21 years

What is this study about?

Neuroblastoma is a rare cancer that starts in early nerve cells, usually in the tummy or around the spine. It mainly affects children under the age of five, though it can occur in older children and young adults too. For many patients, standard treatments like chemotherapy and surgery are very successful. However, in some cases, the cancer does not go away completely or it returns after finishing treatment. When this happens, it is known as relapsed or refractory neuroblastoma, and it can be very difficult to treat.

This clinical trial is exploring whether adding a targeted medicine called bevacizumab to different chemotherapy medicines can help. Chemotherapy works by attacking fast-growing cancer cells directly. Bevacizumab works differently: it blocks a specific protein that tumours use to grow their own blood supply. Without new blood vessels, it is much harder for the tumour cells to survive and spread. The research team wants to find out if combining these approaches works better than using chemotherapy on its own.

Finding better ways to treat neuroblastoma that has returned is one of the most urgent goals in children's cancer research. By taking part, young people help doctors learn which medicine combinations are safest and most effective. The results of this study could help shape standard NHS treatment guidelines for children facing this condition in the future.

Key takeaways

The trial tests if adding bevacizumab to chemotherapy helps treat neuroblastoma that has returned or stopped responding.
It is open to children and young people aged between 1 and 21 years.
Participants are assigned to treatment groups randomly to ensure fair scientific results.
Regular scans and blood tests are used to closely monitor safety and response to treatment.
Taking part is completely voluntary, and you can withdraw at any time.

Who may be eligible?

This trial is open to children, teenagers, and young adults aged between 1 and 21 years who have been diagnosed with neuroblastoma. To take part, the cancer must either have returned after prior treatment (relapsed) or failed to respond adequately to standard frontline therapy (refractory).

Doctors need to be able to see and measure the cancer on hospital scans, such as an MRI, CT, or special MIBG scan. Young people whose cancer can only be detected inside the bone marrow are not able to join this particular study.

Because these treatments are strong, participants must be well enough to cope with them. The hospital team will check that the child's kidneys, liver, and bone marrow are working safely through routine blood and urine tests. A parent, guardian, or the young person themselves (if old enough) must also give written permission to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

The patient is between 1 and 21 years old.
There is a confirmed diagnosis of high-risk neuroblastoma that has returned or not responded to standard treatment.
The tumour can be seen and measured on standard hospital scans.
The cancer is not located only within the bone marrow.
Blood and urine tests show acceptable kidney, liver, and bone marrow function.

Answer every question to see your result.

What does participation involve?

If your child joins the study, a computer will randomly place them into a treatment group to compare different combinations of chemotherapy and bevacizumab fairly. Treatments are given in set cycles, usually lasting a few weeks at a time. Some medicines are taken by mouth at home, while others are given through a drip into a vein at the hospital day unit or during a short ward stay.

Throughout the study, your child will have regular hospital visits for blood tests, urine checks, and physical exams to ensure they stay safe. Every few cycles, doctors will carry out imaging scans (such as CT, MRI, or MIBG scans) to see how the cancer is responding to the medicines.

Treatment continues for as long as it helps your child and any side effects remain manageable. After finishing the trial medicines, your child will have regular follow-up appointments with their care team to check on their recovery, health, and general well-being.

Potential risks and benefits

Taking part in this study may help shrink the tumour or slow its growth, but there is no guarantee of direct benefit. The trial results will also help future children with neuroblastoma. Like all cancer treatments, these medicines can cause side effects. Common ones include tiredness, sickness, higher risk of infections, high blood pressure, and protein leaking into the urine. Your child's care team will monitor them closely and provide medicines to help prevent or ease any discomfort. Joining the trial is entirely voluntary, and you are free to leave at any time without your child's standard NHS care being affected.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

The Royal Marsden NHS Foundation Trust & Institute of Cancer Research
City only
Surrey, England

Common questions

What is bevacizumab and how does it work?

Bevacizumab is a targeted antibody medicine. It stops tumours from building new blood vessels, cutting off the oxygen and nutrients they need to grow.

Will taking part guarantee that the tumour shrinks?

No clinical trial can guarantee success. Doctors hope these combinations are more effective, but the main goal of the trial is to find out if that is truly the case.

How long will my child need to stay in hospital?

Much of the treatment is given during outpatient day visits, but short overnight stays may sometimes be needed depending on the specific medicines and your child's recovery.

Can we leave the study if we change our minds?

Yes. You can withdraw from the trial at any point. Your decision will not change the quality or dedication of your child's NHS care.

Will we have to pay for any of the study medicines?

No. All trial medicines and extra study tests are provided free of charge through the clinical trial programme.

How to find out more

Lucas Moreno

lucas.moreno@vhebron.net 34 93 489 30 00, ext 3093 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.