"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
A trial of dasatinib for children and young people with Philadelphia positive leukaemia (CA180-226)
This trial looked at how well a type of targeted drug called dasatinib worked for children and young people with Philadelphia chromosome positive leukaemia.
The trial was for children and young people up to and including the age of 18. We use the term ‘you’ in this summary, but if you are a parent, we are referring to your child.
These results were published in 2018.
More about this trial
Most people with chronic myeloid leukaemia (CML), and some people with other types of leukaemia have an abnormal chromosome called the
But researchers want to find other treatments. In this trial, they looked at another targeted drug called dasatinib. It is a tyrosine kinase inhibitor and works by blocking a signal that tells leukaemia cells to grow.
We know from research that dasatinib helped adults with CML. Researchers wanted to find out if it could help children and young people who had Philadelphia positive CML.
The aims of this trial were to:
- find out how well dasatinib worked for Philadelphia positive leukaemia in children and young people
- learn more about the side effects in this age group
Summary of results
The trial team found that dasatinib is an acceptable treatment that worked for children and young people with Philadelphia positive leukaemia.
This was a phase 2 trial. Everyone had dasatinib.
To see how well dasatinib worked the team looked at how many leukaemia cells with the Philadelphia chromosome were left in a blood sample after treatment. This is a cytogenetic response.
A complete cytogenetic response is when they can’t see any leukaemia cells with the Philadelphia chromosomes. A partial cytogenetic response is when they can see only a few. The team combined these 2 to find the major cytogenetic response.
There were 3 groups in this trial.
Results for group 1
There were 29 children and young people in this group. They were either having imatinib and it had stopped working. Or they couldn’t have imatinib.
Researchers wanted to know how long it took for more than 30% (30 out of every 100) of the 29 children and young people to have major cytogenetic response. This happened after 3 months of treatment.
At 4 years of treatment 78 out of every 100 (78%) of children and young people in this group were alive and had no sign of their leukaemia.
Results for group 2
There were 17 children and young people in this group who either:
- had acute lymphoblastic leukaemia (ALL) with the Philadelphia chromosome whose imatinib treatment had stopped working or they couldn’t have imatinib
- had chronic myeloid leukaemia (CML) that was in the accelerated or blastic phase whose imatinib treatment had stopped working or they couldn’t have imatinib
This group closed early to recruitment because dasatinib wasn’t working.
Results for group 3
There were 84 children and young people in this group. They were newly diagnosed with Philadelphia positive CML. And had dasatinib as a tablet or liquid.
In this group researchers were looking for when there were no leukaemia cells with the Philadelphia chromosome (a complete cytogenetic response).
They wanted to know how long it took for 55 out of every 100 (55%) children and young people to get a complete cytogenetic response. This happened after 6 months of treatment.
After 4 years of treatment 93 out of every 100 (93%) of children and young people were alive and had no sign of their leukaemia.
The most common side effects reported were the same as for adults who took dasatinib apart from heart problems.
The side effects included:
- feeling or being sick
- muscle or joint pain
The trial team concluded that dasatinib for children and young people with Philadelphia positive CML was well accepted and worked.
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
Professor Pamela Kearns