A trial of clofarabine and liposomal daunorubicin for children and teenagers with acute myeloid leukaemia (CLOUD)
Please note - this trial is no longer recruiting patients. We hope to add results when they are available.
Cancer type:
Status:
Phase:
This trial is looking at clofarabine and liposomal daunorubicin for children and young people who have acute myeloid leukaemia (AML).
This trial is for children and teenagers 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.
Doctors often treat AML with chemotherapy. This usually works very well. But if AML stops responding to treatment, or comes back (relapses) it can be more difficult to treat. Doctors are looking for new combinations of drugs to treat AML that no longer responds to 
.
Clofarabine is a chemotherapy drug that doctors already use to treat children with acute lymphoblastic leukaemia. In this trial, researchers want to see if a combination of clofarabine and another drug called liposomal daunorubicin can help children and young people who have AML.
Liposomal daunorubicin is already sometimes used to treat children with AML. Liposomal drugs are “wrapped up” in a fatty covering called liposome. This helps the drug to work better and causes less severe side effects.
The aims of the trial are to
- Find the highest dose of clofarabine that you can safely have at the same time as liposomal daunorubicin
- Learn more about the side effects
- See if this combination of drugs helps children and teenagers with AML that is not responding to standard chemotherapy
Who can enter
You can enter this trial if you
- Have been diagnosed with AML that is not responding to induction chemotherapy and more than 20% of your bone marrow is made up of undeveloped blood cells (blasts) or
- Have AML that has come back within 1 year, or has come back more than once and more than 5% of your bone marrow is made up of blasts
- Are at least 6 months old, but have not yet reached your 18th birthday
- Are well enough to take part in the trial - this means you have a Karnofsky score of at least 50, or if your child is under 16, the trial team will use another scale to measure how much they can play and how much they need to rest
- Have satisfactory blood test results
- Have recovered from the side effects of any other treatment
- Are willing to use reliable
contraception during the trial if there is any chance you could become pregnant
You cannot enter this trial if you
- Have AML that has come back more than a year after you were first diagnosed
- Have only one area of AML that is outside your bone marrow (isolated extramedullary leukaemia)
- Have AML that has spread to your brain or spinal cord (the central nervous system) and is causing symptoms
- Have had a stem cell transplant in the last 6 months
- Have any signs of a side effect called graft versus host disease after a transplant
- Have not recovered from other side effects of a transplant
- Have already had a large dose of daunorubicin or a similar drug (the trial doctors can advise you about this)
- Have had chemotherapy in the last 2 weeks
- Have had monoclonal antibody treatment in the last 2 weeks
- Are having any other treatment for AML, or any other experimental drug as part of another clinical trial
- Have certain heart problems (the trial doctors can advise you about this)
- Have an infection or any other serious medical condition
Trial design
This is a phase 1 trial. It will recruit between 12 and 18 people. Everybody taking part will have clofarabine and liposomal daunorubicin. The first patients taking part will have the lowest dose of clofarabine. If they don’t have any serious side effects, the next patients will have a higher dose. And so on, until they find the best dose to give. This is called a ‘dose escalation study’. Everybody will have the same dose of liposomal daunorubicin.
You have clofarabine every day for 5 days through a drip into a vein. You will probably have this via a central line or portacath. It takes 2 hours each time. On days 1, 3 and 5, you also have liposomal daunorubicin through the drip. This starts about 4 hours after the clofarabine and also takes about 2 hours.
If the trial treatment helps you, the doctors may talk to you about having other treatments such as a bone marrow transplant.
Hospital visits
You will see the doctors and have some tests before you start treatment. The tests include
- Physical examination
- Blood tests
- Bone marrow test
- Heart trace (
ECG ) - Heart scan (
echocardiogram ) - Pregnancy test (if you are female and could possibly become pregnant)
You will be in hospital for at least 5 days to have this treatment. You have more blood tests at the end of the first week and then each week for the next month. You have another ECG and echocardiogram between 3 and 4 weeks after starting treatment, and another bone marrow test between 3 and 6 weeks after starting. You see the trial doctors at the end of 6 weeks and then once a month after that. You will have blood tests each time.
Side effects
As this is a new combination of drugs, there may be some side effects that we don’t know about yet. The side effects of liposomal daunorubicin and clofarabine that are known include
- A drop in the number of blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness
- Sickness
- Diarrhoea
- Sore mouth
- Hair loss
- Headache
- Tiredness (fatigue)
- Weakness
- Shivering
- Breathlessness
- Changes to the heart
There is more information about the side effects of chemotherapy on this website.
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.
Chief Investigator
Professor Pamela Kearns
Supported by
Bloodwise
Cancer Research UK Clinical Trials Unit University of Birmingham
Children's Cancer and Leukaemia Group (CCLG)
Experimental Cancer Medicine Centre (ECMC)
Genzyme Co
NIHR Clinical Research Network: Cancer
If you have questions about the trial please contact our cancer information nurses
Freephone 0808 800 4040
