"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 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.
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
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
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 (
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
- 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 marrowin 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 systemsystem 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
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.
You see the trial team and have some tests before you start treatment. The tests include
- Physical examination
- Blood tests
- Heart trace (
- Heart ultrasound (
- 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.
The side effects of nilotinib include
- Feeling or being sick
- Constipation or Diarrhoea
- Tiredness (fatigue)
- Skin changes such as itching, skin rash, or dry skin
- Hair loss
- High temperature (fever)
- Shortness of breath
- A drop in the number of red blood cells (
- A build up of fluid in your body (oedema)
- Pain in your arms, legs or tummy (abdomen)
We have more information about nilotinib.
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.
Professor Pamela Kearns
NIHR Clinical Research Network: Cancer