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)

Leukaemia

Status:

Results

Phase:

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:

just been diagnosed with chronic myeloid leukaemia (CML)
CML that wasn’t responding to drugs called imatinib or dasatinib
acute lymphoblastic leukaemia (ALL) that had got worse or came back despite having other treatment

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 . 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

Results
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

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 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.