A trial of dasatinib for children and young people with Philadelphia positive leukaemia (CA180226)

This trial is looking at dasatinib to treat children and young people who have Philadelphia chromosome positive leukaemia. It is for people who have either just been diagnosed, or have leukaemia that has not responded to imatinib.

The trial is for children and young people up to and including the age of 18. We use the term ‘you’ in this summary, but of course if you are a parent, we are referring to your child.

Most people with chronic myeloid leukaemia (CML), and some people with other types of leukaemia have an abnormal chromosome called the Philadelphia chromosome. You may hear this called ‘Philadelphia positive leukaemia’.

Doctors can treat Philadelphia positive leukaemia with a type of biological therapy called imatinib (Glivec).

But researchers want to find other treatments to use if imatinib doesn’t help, or if leukaemia stops responding to it. In this trial, they are looking at another biological therapy drug called dasatinib. It is called a tyrosine kinase inhibitor and works by blocking a signal that tells leukaemia cells to grow.

We know from research that dasatinib can help adults with CML. Researchers want to see if it can help children and young people who have CML or other types of Philadelphia positive leukaemia.

The aims of this trial are to

Find out how well dasatinib works for Philadelphia positive leukaemia in children and young people
Learn more about the side effects in this age group

Recruitment

Start 01/12/2009

End 30/11/2012

Phase

Phase 2

Who can enter

You can enter this trial if you

Are at least 1 year old, but have not yet reached your 19th birthday
Have chronic myeloid leukaemia (CML) or acute lymphoblastic leukaemia (ALL) that is Philadelphia positive
Have leukaemia that has just been diagnosed and you haven't had any treatment yet, or it has not responded to imatinib, has stopped responding to it, or you could not take imatinib because you had bad side effects
Have satisfactory blood test results
Are well enough to take part in the trial (Karnofsky score higher than 50)
Have recovered from the side effects of other treatment unless they are very mild (you can take part if you still have hair loss)
Are willing to use reliable contraception (if applicable) during the trial and for 3 months afterwards if there is any chance you or your partner could become pregnant

You cannot enter this trial if

Your doctors think there is another treatment that could cure your leukaemia
Leukaemia cells have spread into your brain or spinal cord (central nervous system) and this is causing symptoms
You have already had dasatinib
You have had treatment as part of another clinical trial in the last 2 weeks
You have an infection that cannot be controlled, bleeding problems, heart problems, or any other serious medical condition that could affect you taking part
You take any medication that can affect your heart or how your blood clots – the trial doctors can advise you about this
You are pregnant or breastfeeding

Trial design

This is an international study. It will recruit about 110 people in different countries. Everybody taking part will have dasatinib.

You take dasatinib as a tablet. But if you have problems swallowing tablets, you can have it as a liquid, dissolved in apple or orange juice.

You have dasatinib for at least 2 years. Whether or not you take it for longer depends on how well the treatment is working, and whether you have any side effects.

Hospital visits

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

Physical examination
Heart trace (ECG)
Heart scan (echocardiogram)
Chest X-ray
Blood and urine tests
Bone marrow test
Bone growth assessments

You may also need to have a lumbar puncture.

During treatment, you will see the doctors regularly. To begin with, this will be once a week, then once a month, and eventually every 3 months. You will have an ECG after 2 weeks of treatment, and other scans or X-rays if your doctor thinks you need them. If you have just been diagnosed with CML, you will have an ECG and echocardiogram every year during the trial. As part of the bone growth assessments you have as wrist X-ray and bone scan (DXA scan) every year.

You will have blood tests

Every week for the first 6 weeks (you may need to have blood tests more often)
Monthly for the next 6 months,
Then every 3 months for 2 years
Every 6 months after that

The doctors will take extra blood samples for 'molecular analysis’ every 3 months for the first 2 years and every 6 months after that. This is so that they can study your genes.

Throughout the trial, you will need to have bone marrow tests every 3 months to find out if the treatment is having an effect on your leukaemia.

If you are female and of a child bearing age, you will have a pregnancy test before you start treatment and then every 2 months during the trial.

At the end of the trial you will see the research doctors and have more tests. If you are still having side effects from the treatment, you will see them every 4 weeks until these get better. You will then see them every year for 5 years. You will have bone growth assessments to find out if dasatinib has any effect on your long term bone growth and development. This includes blood and urine tests, a wrist X-ray and a DXA scan.

Side effects

The side effects of dasatinib include

A drop in blood cells counts causing an increased risk of infection, tiredness, shortness of breath, bruising and bleeding problems
Sickness
Diarrhoea
Loss of appetite
Headache
Tiredness (fatigue)
A build up of fluid in different parts of the body (fluid retention)
Rash
Shortness of breath or cough
Muscle or bone pain