A trial looking at chemotherapy for children and young people with advanced neuroblastoma (NB 2006 05)

Cancer type:

Children's cancers




Phase 2

This trial was looking at a new combination of chemotherapy Open a glossary item drugs. It was for children with neuroblastoma whose treatment had not worked as well as doctors had hoped.

More about this trial

Neuroblastoma is a rare type of cancer that develops in nerve cells, most commonly in nerve cells of the adrenal glands Open a glossary item. It usually affects babies and young children. Neuroblastoma can spread to other parts of the body. This is called advanced cancer Open a glossary item. The most common places for neuroblastoma to spread are the bones, liver and skin. 

Children with neuroblastoma usually have a combination of treatments including surgery Open a glossary item, chemotherapy and radiotherapy Open a glossary item. Treatment depends on:

  • the stage Open a glossary item of neuroblastoma 
  • the risk group - if there is a low, medium or high risk of the cancer coming back after treatment

Sometimes the treatment doesn’t work very well, or the cancer comes back after treatment (recurs). If this happens it is more difficult to treat.

Vincristine and doxorubicin are standard chemotherapy drugs Open a glossary item used to treat children and young people with cancer. Topotecan is a chemotherapy drug used to treat some adult types of cancer. And it’s already been looked at in trials for some childhood cancers. Earlier studies in childhood cancers had shown that adding topotecan to other drugs could help treatment work better.

This trial aimed to find out:

  • if treatment with topotecan, vincristine and doxorubicin (also known as TVD) could shrink neuroblastoma
  • about the side effects of TVD

The trial was for children over 1 year and young people under the age of 20 with advanced neuroblastoma. They were at high risk of their cancer coming back after treatment. And their first line chemotherapy Open a glossary item hadn’t got rid of or shrunk their neuroblastoma very well.

Summary of results

The trial team found that TVD helped some children and young people with advanced, high risk neuroblastoma.

63 children and young people took part. Everyone had TVD. They had treatment in 3 week periods called cycles of treatment

They had topotecan on days 1 to 5 through their central line. This was followed by vincristine and doxorubicin non stop through a pump over days 5 and 6. They had no treatment in weeks 2 or 3. After the first cycle of treatment the children and young people had tests to find out if treatment had worked. 

Everyone had a good enough response so they all had another cycle of treatment over 6 weeks. After the second cycle of treatment, everyone had tests to find out if treatment had continued to work.

The trial team analysed the test results after 2 courses of TVD and:

  • the cancer went away in 4 children and young people– doctors call this a complete response Open a glossary item 
  • in 28 the cancer got smaller – this is called a partial response Open a glossary item
  • in 16 children and young people, where the neuroblastoma had spread to other parts of the body, these areas had got smaller
  • in 14 children and young people the cancer stopped growing (doctors call this stable disease Open a glossary item)

The neuroblastoma continued to grow during the TVD treatment for 1 child taking part. 

For 23 out of the 63 young people, the TVD worked well enough to have the next phase of their treatment plan. This is called high dose treatment.

The researchers also found that TVD worked better for neuroblastoma that had spread to the bone marrow Open a glossary item than to bone. And it didn’t work as well for those with neuroblastoma that stayed the same, or got worse, after first line chemotherapy.

Side effects

The trial team also looked at the side effects Open a glossary item of TVD. The main side effects were:

The trial team concluded that TVD can help children and young people with advanced neuroblastoma. They recommend more clinical trials comparing TVD with other first line chemotherapy treatments.

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 G. Makin

Supported by

Cancer Research UK Children's Cancer Trials Team
University of Birmingham
NIHR Clinical Research Network: Cancer

If you have questions about the trial please contact our cancer information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:


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

Charlie took part in a trial to try new treatments

A picture of Charlie

“I think it’s really important that people keep signing up to these type of trials to push research forward.”

Last reviewed:

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