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

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Acute leukaemia

Acute lymphoblastic leukaemia (ALL)

Blood cancers

Children's cancers

Chronic leukaemia

Chronic myeloid leukaemia (CML)

Leukaemia

Status:

Closed

Phase:

Phase 1

This trial is looking at nilotinib for children and young people who have Philadelphia positive leukaemia. It is for children who have just been diagnosed with chronic myeloid leukaemia (CML), or have CML that is not responding to drugs called imatinib or dasatinib, or have acute lymphoblastic leukaemia (ALL) that has got worse or come back despite having other treatment.

The trial is 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.

Most people with CML, and some people with other types of leukaemia have an abnormal chromosome Open a glossary item called the Philadelphia chromosome . You may hear this called Philadelphia positive leukaemia.

Doctors can treat newly diagnosed chronic myeloid leukaemia (CML) in adults with biological therapy drugs called imatinib and nilotinib. They can also use nilotinib if CML doesn’t respond to imatinib or becomes resistant Open a glossary item to it, or for people who can't take imatinib because they have bad side effects.

Researchers want to see if nilotinib could also help children and young people who have Philadelphia positive leukaemia. The aim of this study is to learn more about what happens to nilotinib in your body (pharmacokinetics Open a glossary item ).

Who can enter

You may be able to enter this trial if you

Are under 18 and have recently been diagnosed with chronic myeloid leukaemia (CML), or have CML that has got worse despite having imatinib or a drug called dasatinib (or you can’t take these drugs because of bad side effects) and is in chronic or accelerated phase or you have acute lymphoblastic leukaemia (ALL) that has got worse or come back despite having standard treatment
Have leukaemia that has the Philadelphia chromosome (Philadelphia positive)
Have satisfactory blood test results
Are well enough to take part (children under 10 must have a Lansky scale score of 50 or more)

You cannot enter this trial if you

Have a change to a gene called T3151 – you can check this with your doctor
Have a drug called dasatinib in the 3 days before starting the trial treatment, or imatinib in the 5 days before
Have a growth factor called Neulasta (a type of GCSF) in the 2 weeks before starting the trial treatment or other growth factors for blood cells in the week before
Have chemotherapy that could affect your bone marrow in the 2 weeks before you start the trial treatment
Have not recovered from the side effects of earlier chemotherapy (apart from hair loss) unless they are very mild
Have had more than 2 weeks of treatment with hydroxyurea for CML or steroids for ALL and this treatment cannot be stopped within a week of starting the trial treatment
Have had a stem cell transplant in the last 3 months and you have signs of graft versus host disease (your doctor can confirm this)
Have had radiotherapy in the last 6 weeks that included a large part of your bone marrow, radiotherapy to your brain and spine or to your whole body (TBI) in the last 3 months, or radiotherapy for symptoms in the last 2 weeks
Take drugs that could affect your heart rhythm or drugs that can affect an enzyme called CYP3A4 and you cannot stop taking them or change to another drug at least 2 weeks before you start the study treatment – it is important that you don’t stop taking any medication without speaking to your doctor
Have sickness or diarrhoea that cannot be controlled with medication, or other problems with your digestive system system that could affect how you absorb tablets
Have had pancreatitis (inflammation of the pancreas)
Have problems with your kidneys, liver or pancreas that are not related to your leukaemia
Have a serious infection or heart problems that are a cause for concern (the trial doctors can advise you about this)
Are known to be HIV, hepatitis B or hepatitis C positive
Are known to be very sensitive to anything in nilotinib, including lactose which is a sugar found mainly in milk

Trial design

This is a phase 1 trial that will recruit up to 14 children and young people. Everybody taking part has nilotinib. Children under 1 year old cannot take part.

Everybody joining the trial takes nilotinib capsules by mouth.

On the first day of treatment you have just 1 dose of nilotinib. After that, you take nilotinib twice a day. You take the capsules every day, but researchers call each 4 week period a cycle of treatment.

If you don’t have any bad side effects, you can have up to 12 cycles of treatment, which takes nearly a year.

Hospital visits

You see the trial team and have some tests before you start treatment. The tests include

Physical examination
Blood tests
Heart trace (ECG )
Heart ultrasound (echocardiogram )
Bone marrow test
Lumbar puncture (if you have ALL)

You go to hospital

Once a week in the first cycle of treatment
Every 4 weeks during the next 11 treatment cycles

You have blood tests at each hospital visit and an ECG at least once in each treatment cycle. If you have only just been diagnosed with CML, you have an extra ECG after 4 days of treatment.

If you have ALL, you have a bone marrow test every 6 months

When you finish the treatment, a member of the trial team will contact you or your doctor about a month later to see how you are and whether you have any long term side effects. They may then phone you (or your doctor) every 6 months to see how you are.