A study looking at a new biological therapy to treat 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)

Leukaemia

Status:

Closed

Phase:

Phase 2

This study is to see how well nilotinib (Tasigna) works as a first treatment for chronic myeloid leukaemia (CML). Most people with chronic myeloid leukaemia (CML) have an abnormal chromosome Open a glossary item called the Philadelphia chromosome. This develops if some DNA from one chromosome sticks to another when the cell divides, creating an abnormal gene called ‘bcr/abl’. Bcr/abl produces an enzyme called tyrosine kinase , which encourages leukaemia cells to grow and multiply.

Doctors usually treat CML with a biological therapy called imatinib. Imatinib is a tyrosine kinase inhibitor. It switches off bcr/abl, stopping production of tyrosine kinase.

This study is looking at a new drug called nilotinib. Nilotinib is very similar to imatinib. Doctors think nilotinib may be better than imatinib at blocking tyrosine kinase. We know from research that this works well to treat CML if you are no longer able to take imatinib.

Doctors want to find out more about nilotinib as a first treatment for CML. And see if it works as well at a lower dose than they have used before. The main aims of this study are to see how well a reduced dose of nilotinib

Works to treat CML
Reduces the side effects of this treatment

Who can enter

You can enter this study if you

Have chronic myeloid leukaemia (CML) which is stable and only developing slowly (chronic phase)
Have had CML for less than 6 months
Have tested positive for the Philadelphia chromosome
Have satisfactory blood tests
Are well enough to take part (performance status 0, 1 or 2)
Are willing to closely follow the study team’s instructions
Are willing to use reliable contraception throughout the study if there is any chance that you or your partner could become pregnant
Are at least 18 years old

You cannot enter this study if you

Have CML that has spread from your bone marrow to other organs in your body (‘extramedullary disease’) - if you have a swollen liver or spleen ‘hepatosplenomegaly’ you can still take part
Have a change to a gene called T3151 – you can check this with your doctor
Have had any treatment for CML before (if you have taken imatinib for less than 2 weeks, or hydroxyurea or anagrelide for less than a month you may still be able to take part)
Have any other cancer unless this does not need to be treated at the moment, or your doctor does not think it will affect the study
Have a bleeding disorder that is not related to your CML
Have had a heart attack in the last year, or have any other heart condition that is a cause for concern
Have ever had long term (chronic) pancreatitis or have had acute pancreatitis in the last year
Have any problems that would make it difficult for you to absorb tablets
Have had short or long term liver problems, or severe kidney disease that is not related to your CML
Have any other severe or uncontrolled medical condition, for example uncontrolled diabetes, or an uncontrolled infection
Have had surgery but you have not yet fully recovered
Are having treatment with a drug that affects an enzyme in the body called CYP3A4 – you can check this with your doctor
Are taking medication that may cause an abnormal heart rhythm, but you are not able to stop this or swap to a different one before the study – you can check this with your doctor
Have had any other trial drug in the month before you would start the study
Are pregnant or breastfeeding

Trial design

This international study will recruit 60 people. You take nilotinib twice a day for up to 2 years. You take the capsules in the morning and evening. You swallow them with a full glass of water. For 2 hours before and one hour after each dose, you must not eat food or drink anything other than water.

You must also avoid eating grapefruit, orange or star fruit (or drinking these juices) during this study as they can affect how well the drugs work.

Hospital visits

Before you start the study, you will see the doctor and have some tests, including

Blood tests and urine tests
Bone marrow test (if needed)
Heart trace (ECG)
Heart scan (Echocardiogram)

You will have a blood test each week for the first month that you take nilotinib. You will also have an ECG at 2 of these visits.

You then have a blood tests every month while you are taking nilotinib. You may have more ECGs during the study if your doctor thinks you need them.

You will also have a bone marrow test every 3 months until all the blood cells in your bone marrow are normal.

At the end of the study you repeat the tests you had before you started.

Side effects

Common side effects of nilotinib include

Feeling or being sick
Constipation or diarrhoea
Tiredness
Headache
Pain in tummy (abdomen), bones, joints and muscles
Loss of appetite
Changes in weight
Hair loss
Heavy sweating
Dizziness
Numbness or tingling
A rash or dry skin
A drop in the number of blood cells leading to an increase risk of infection, tiredness, shortness of breath, bruising and bleeding problems
Short term changes to the way your liver works
Rapid heart beat