"I am glad that taking part in a trial might help others on their own cancer journey.”
A trial of decitabine followed by cytarabine for children and teenagers with acute myeloid leukaemia that is not responding to treatment or has come back
Please note - this trial is no longer recruiting patients. We hope to add results when they are available.
This trial is looking at decitabine followed by cytarabine for acute myeloid leukaemia that isn’t responding to treatment or has come back afterwards.
More about this trial
The trial is recruiting children and young people up to and including the age of 17. We use the term ‘you’ in this summary, but of course if you are a parent, we are referring to your child.
Children with acute myeloid leukaemia (AML) usually have chemotherapy. This can work well, but sometimes the leukaemia doesn’t respond to treatment, or it comes back later on. Researchers are looking for treatments to help in this situation. In this trial, they are looking at a drug called decitabine.
Doctors can already use decitabine to treat AML in people over 65. In this trial, they want to see if it helps people under 18 who have AML. The children taking part will have decitabine, followed by a drug called cytarabine that doctors can already use in chemotherapy to treat leukaemia in children.
The aims of the study are to
- Find which of 3 doses of cytarabine is the highest safe dose that children under 18 can have safely after having decitabine
- See if having decitabine before cytarabine helps children with AML that isn’t responding to treatment or has come back
Who can enter
You may be able to join this trial if all of the following apply. You
- Are at least 1 month old but have not yet reached your 18th birthday
- Have acute myeloid leukaemia (AML) that has not responded to
standard treatmentor has come back afterwards (including after a bone marrow transplant), and there is no other treatment available that would aim to cure your leukaemia
- Are well enough to take part
- Have recovered from the side effects of any other treatment you’ve had
- Are willing to use reliable contraception if you are sexually active and there is any chance you or your partner could become pregnant
You cannot join this trial if any of these apply. You
- Have already had decitabine or a drug called azacitidine
- Have a type of AML called acute promyelocytic leukaemia
- Have leukaemia that has spread to your brain or spinal cord and this requires treatment directed at your brain or spinal cord
- Have leukaemia that is associated with Down’s syndrome or certain other inherited syndromes (the trial team can advise you about this)
- Are having another experimental treatment as part of a trial
- Are pregnant or breastfeeding
This is a phase1/2 trial. The researchers need about 33 children and teenagers to join. Everybody taking part will have decitabine and cytarabine through a drip. The drip is attached to a line that goes into a large vein. This is called a central line.
In the 1st part of the trial, the researchers will test 3 different doses of cytarabine. They want to find the highest dose that people under 18 can have safely after having decitabine. In the 2nd part of the trial, everybody will have the highest safe dose of cytarabine found in part 1.
You have treatment in 4 week periods called cycles of treatment. In each cycle of treatment you have
- Decitabine on days 1 to 5 – this takes about an hour each time
- Cytarabine on days 8 to 12 – this takes about 4 hours each time
Having one drug followed by the other is called sequential treatment. You have up to 4 cycles of sequential treatment.
If after finishing the sequential treatment, your doctor thinks you would benefit from having more decitabine, you may be able to carry on having it on its own for as long as it helps you.
You see the trial team and have some tests before you start treatment. The tests include
- Physical examination
- Blood tests
- Bone marrow test
- Heart scan (
echocardiogram) to check the functioning of your heart
You see the trial team and have blood tests regularly throughout your treatment. The trial team will tell you more about this.
You have a bone marrow test after 4 weeks of treatment. And you may need to have another one 4 weeks later.
When you finish treatment, you see the trial team again about a month later. After this, they will continue to check how you are every 3 months until the trial has completely finished. But you will not necessarily have to go to hospital for this.
Doctors don’t usually use decitabine to treat children so there may be side effects we don’t know about yet. In adults the most common side effects include
- A drop in blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness
- Swollen ankles
- Feeling or being sick
- Constipation and tummy (abdominal) pain
- Loss of appetite
You will have had cytarabine as part of your earlier chemotherapy. But the trial team will talk to you about all the possible side effects and explain when to contact them if you have any of these.
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 Pam Kearns
Janssen-Cilag International NV
NIHR Clinical Research Network: Cancer