Last year in the UK over 60,000 cancer patients enrolled on clinical trials aimed at improving cancer treatments and making them available to all.
A trial of ipilimumab for children and teenagers who have melanoma that can't be removed with surgery (CA184178)
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
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.
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.
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
- problems with how the
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 (
How to join a clinical trial
Dr Julia Chisholm
NIHR Clinical Research Network: Cancer