A trial looking at nilotinib for newly diagnosed chronic myeloid leukaemia

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

Cancer type:

Blood cancers
Chronic leukaemia
Chronic myeloid leukaemia (CML)




Phase 3

This trial is looking at nilotinib for people with newly diagnosed chronic myeloid leukaemia, who are Philadelphia chromosome positive, or have a high level of a protein called BCR-ABL (or both) and whose leukaemia is in chronic phase.

Doctors usually treat chronic myeloid leukaemia (CML) with a type of biological therapy called imatinib. This often works well. But sometimes CML doesn’t respond to imatinib or becomes resistant to it. And some people cannot take it because they have bad side effects. In this situation, you may have a drug called nilotinib. Nilotinib works in a similar way to imatinib, but may have more of an effect on the BCR-ABL protein.

In this study, researchers are trying to find out more about having nilotinib as the first treatment after being diagnosed with CML. And they will look at the effect it has on the amount of Philadelphia chromosome and abnormal proteins in your blood. This is because the more the levels go down, the more likely you are to have a good, long term response to treatment.

The aims of this trial are to

  • See if CML in chronic phase responds better to nilotinib as first line treatment than it does to imatinib
  • Learn more about the different ways people respond to treatment

Who can enter

You can enter this trial if you

  • Have been diagnosed with chronic myeloid leukaemia (CML) in the last 6 months
  • Haven’t yet had any treatment, apart from a drug called hydroxyurea or imatinib (as long as you have not been taking it for more than 3 months)
  • Have CML that is Philadelphia chromosome positive, or BCR-ABL positive (or both) – your doctor can advise you about this
  • Have satisfactory blood test results
  • Are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
  • Are willing to use reliable contraception during the trial and for 3 months afterwards if you are a woman (4 weeks if you are a man) if there is any chance you or your partner could become pregnant
  • Are at least 18 years old

You cannot enter this trial if you

  • Have taken hydroxyurea for more than 6 months, or imatinib for more than 3 months
  • Have had surgery in the last 2 weeks, or have not recovered from earlier surgery
  • Have had treatment with a growth factor that affects blood cells in the last week
  • Have any other type of cancer that is causing symptoms or needs treatment at this time
  • Have had a heart attack in the last year, or you have heart pain (angina), heart failure, high blood pressure that cannot be controlled with medication, or any other heart problem that is a cause for concern
  • Have had pancreatitis (inflammation of the pancreas)
  • Have any other serious medical condition that the trial doctors think could affect you taking part in the study
  • Have any problems that would make it difficult for you to absorb tablets
  • Need to carry on taking any other drugs that can affect your heart rhythm, or affect CYP enzymes (your doctor can advise you about this - it is important that you don’t stop taking any sort of medication before discussing it with your doctor)
  • Are HIV positive
  • Are pregnant or breastfeeding

Trial design

The trial will recruit over 800 people in Europe, including about 30 in the UK. Everybody taking part will have nilotinib capsules twice a day. You have to swallow them with a glass of water. You must not eat or drink anything other than water for 2 hours before taking the capsules or for 1 hour afterwards.

The study lasts for 2 years. But if nilotinib is helping you, it will be provided free of charge for up to 6 years after the end of the study.

Hospital visits

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

  • Physical examination
  • Heart trace (ECG)
  • Blood tests

You may also have a bone marrow test, unless you have already had one in the last 8 weeks.

You will see your study doctor every 3 months for up to 2 years. You have a physical examination and blood tests at each visit. You also have

  • Bone marrow tests after 3, 6, 12 and 18 months
  • ECG s after 1, 3, 6, 12 and 18 months

You will see the trial team again and have tests again at the end of 2 years, or sooner if you stop taking nilotinib for some reason.

Side effects

As nilotinib is quite a new drug, there may be side effects we don’t know about yet. The known side effects include

  • Feeling or being sick
  • Fever (high temperature)
  • Diarrhoea or constipation
  • Rash, itching or dry skin
  • Tiredness (fatigue)
  • Cough
  • Headache
  • Loss of appetite
  • A drop in the number of red blood cells (anaemia) causing tiredness or shortness of breath
  • Fluid retention (oedema)
  • Pan in the abdomen or limbs

There is more information about the possible side effects of nilotinib on CancerHelp UK.

You must not eat certain fruits such as grapefruit, pomegranate, Seville oranges (or drink these juices) during this trial as they can affect how the drugs work and make side effects worse.

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

Dr David Marin

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Last review date

CRUK internal database number:

Oracle 7274

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Over 60,000 cancer patients enrolled on clinical trials in the UK last year.

Last reviewed:

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