A trial of ipilimumab for children and teenagers who have melanoma that can't be removed with surgery (CA184178)

Cancer type:

Children's cancers
Skin cancer




Phase 2

This trial looked at ipilimumab to improve treatment for children and young people who have melanoma.

It was for people who couldn’t have surgery to remove the melanoma or it had spread elsewhere in the body. Melanoma that has spread is advanced melanoma. 

This trial was open for people to join between 2013 and 2016. The team published the results in 2017.

More about this trial

It is usual to have surgery to remove melanoma skin cancer but sometimes this isn’t possible because the cancer has spread. 

Researchers are always looking at ways to improve treatment for melanoma in children and teenagers. In this trial they wanted to find out if an immunotherapy called ipilimumab would be useful for people in this situation. 

Ipilimumab helps the immune system Open a glossary item to find and kill cancer cells. When this trial was done it was already a treatment for advanced melanoma in adults. 

The main aims of this trial were to see:

  • how well ipilimumab works in children and teenagers who have advanced melanoma
  • what the side effects are

Summary of results

The trial team found that ipilimumab worked for some children and teenagers with advanced melanoma. 

Trial design
This trial included children and teenagers between the ages of 12 and 18.

It was a small phase 2 trial. The team had hoped to find 40 people to take part but only 12 children and teenagers joined. 

There were 2 doses of ipilimumab:

  • 4 people had a lower dose - they had about 3 doses in total 
  • 8 people had a higher dose – they had about 4 doses in total 

The team looked at whose melanoma responded to treatment. 

4 people had the lower dose. The team found the melanoma:

  • stayed the same in 1 person
  • continued to grow in 3 people

8 people had the higher dose. The team had the results for 6. They found that the melanoma:

  • got a bit smaller in 2 people (a partial response)
  • continued to grow in 4 people

At 3 years 1 person in the higher dose group still had a partial response. They hadn’t needed any further treatment. 

The trial team also looked at the number of people living. At 1 year this was:

  • 3 out of 4 people who had the low dose
  • 5 out of 8 people who had the high dose

The trial had to close early as it was difficult to find enough children and teenagers to take part. 

Side effects
Most people had at least one side effect. 

People who had the higher dose of ipilimumab had worse side effects. Some of these included:

  • liver changes
  • fevers
  • problems with how the pancreas Open a glossary item  worked 

Some had their treatment stopped due to side effects. This happened to:

  • 1 person in the lower dose group
  • 5 people in the higher dose group

The side effects were similar to the side effects that adults who take ipilimumab have. No one had any new or unexpected side effects. 

The trial team found that ipilimumab worked for some children and teenagers who have advanced melanoma. And the side effects were similar to those that adults have.

This was a small trial as it was difficult to find enough people to join. The team say this highlights the difficulties of finding children and young people with rare cancers to join trials. They suggest that these groups are considered for entry into adult trials looking at promising drugs. 

Where this information comes from    
We have based this summary on information from the research team. The information they sent us has been reviewed by independent specialists (peer reviewed Open a glossary item) and published in a medical journal. The figures we quote above were provided by the trial team who did the research. We have not analysed the data ourselves.

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 Julia Chisholm

Supported by

Bristol-Myers Squibb
NIHR Clinical Research Network: Cancer

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.

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

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