A trial of nilotinib for children and young people with Philadelphia positive leukaemia

Cancer type:

Acute leukaemia
Blood cancers
Children's cancers
Chronic leukaemia
Chronic myeloid leukaemia (CML)




Phase 1

This trial looked at nilotinib for children and young people whose leukaemia had the Philadelphia chromosome Open a glossary item. 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.

More about this trial

Most people with CML, and some people with other types of leukaemia have an abnormal chromosome Open a glossary item. This is the Philadelphia chromosome Open a glossary item. Doctors call this Philadelphia positive leukaemia.

Doctors can treat CML in adults with the targeted cancer drugs Open a glossary item 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 (pharmacokinetics Open a glossary item).

Summary of results

The study team found that children and young people could have the same dose of nilotinib as adults. 
Study design
This was a phase 1 Open a glossary item study. A total of 15 children and young people aged between 5 and 17 years old took part. They all had Philadelphia positive leukaemia:

  • 11 had CML 
  • 4 had ALL

The team took blood samples at regular times to work out how:

  • much nilotinib the body absorbed
  • the body breaks down nilotinib 
  • much nilotinib wasn’t used by the body

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 gene Open a glossary item 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:

  • headache
  • being sick
  • an increase of bilirubin in the blood
  • a skin rash

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 phase 2 Open a glossary item 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 (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

Professor Pamela Kearns

Supported by

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.

Wendy took part in a new trial studying the possible side effect of hearing loss

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"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”

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