
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.
This trial looked at nilotinib for children and young people whose leukaemia had the . It was for children who had:
The trial was for children and young people up to the age of 18. We use the term ‘you’ in this summary, but if you are a parent, we are referring to your child.
This trial was open for people to join between 2011 and 2014. The team published the results in 2020.
Most people with CML, and some people with other types of leukaemia have an abnormal . This is the
. Doctors call this Philadelphia positive leukaemia.
Doctors can treat CML in adults with the imatinib and nilotinib. They also use nilotinib for CML if imatinib stops working. Also, people who can't take imatinib because they have bad side effects may be offered it.
Researchers wanted to see if nilotinib could help children and young people who have Philadelphia positive leukaemia. The aim of this study was to learn more about what happens to nilotinib in your body ().
The study team found that children and young people could have the same dose of nilotinib as adults.
Study design
This was a study. A total of 15 children and young people aged between 5 and 17 years old took part. They all had Philadelphia positive leukaemia:
Results
The team took blood samples at regular times to work out how:
The team compared the results with how adults bodies dealt with nilotinib. The found there was no, or very little difference in the way children’s and young people’s bodies dealt with nilotinib.
They also looked at how well nilotinib worked.
For 3 children and young people with CML a blood test showed that there was a smaller number of the BCR-ABL in their blood. This is a major molecular response and that shows nilotinib helped.
For 3 children and young people with ALL there was no sign of their leukaemia. This is a complete response.
Side effects
Nilotinib was generally well tolerated.
The most common side effects reported were:
Conclusion
The team concluded that children and young people with CML and Philadelphia positive ALL can have the same dose as adults.
They recommended doing a study. We have information about this study.
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 () 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.
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Professor Pamela Kearns
NIHR Clinical Research Network: Cancer
Novartis
Freephone 0808 800 4040
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.