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

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 

ca184178_trial_diagram.png

Results
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   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. 

Conclusion
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 ) 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.