"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
A trial looking at treatment for older people with acute myeloid leukaemia and high risk myelodysplastic syndrome (AML18)
This trial is looking at treatment for acute myeloid leukaemia (AML) and high risk myelodysplastic syndrome (MDS). It is designed mainly for people over 60 years of age who are fit enough to have intensive chemotherapy. But some younger people may be able to take part.
The trial is supported by Cancer Research UK.
More about this trial
The trial is trying to answer a number of questions about the treatment of acute myeloid leukaemia (AML) and high risk myelodysplastic syndrome (MDS). High risk MDS means that there is more chance that it will turn into AML over time. MDS is classed as high risk if more than 10% of your
Doctors usually treat AML with chemotherapy to get rid of the leukaemia cells (getting it into
But a trial called AML 16 showed that adding a drug called gemtuzumab ozogamicin reduced the risk of AML coming back. Gemtuzumab ozogamicin (also known as Mylotarg) is a monoclonal antibody that is attached to a chemotherapy drug called calicheamicin. In the AML 16 trial, people had 1 dose of Mylotarg. In this trial, researchers want to see if having 2 doses of Mylotarg is better than 1.
Doctors can look closely at the
The trial will also look at adding a drug called quizartinib to chemotherapy to see if this helps to control leukaemia or MDS for longer. Quizartinib is a type of biological therapy that can stop signals cancer cells use to divide and grow.
Another thing being looked at in this trial is the best treatment for people who have no leukaemia cells showing in their blood or bone marrow after the 1st cycle of chemotherapy (a complete remission). Even with a complete remission, there may still be some leukaemia cells left. This is called minimal residual disease or MRD. Doctors can use different tests to look for MRD. If they show there is residual disease, you may need more treatment.
The trial aims to find out
- If 2 doses of Mylotarg with induction chemotherapy is better than 1 dose for people with good or intermediate risk cytogenetics
- If either a short course or a long course of a drug called quizartinib improves treatment
- If knowing whether or not there is minimal residual disease (MRD) after 1 cycle of chemotherapy is helpful, and if the outcome of treatment can be improved by making it more intensive for people who do still have MRD after the 1st cycle
- Whether having another treatment cycle of DA works better for people who have MRD after 2 treatment cycles of DA chemotherapy
- Whether vosaroxin and decitabine work for people with known adverse risk cytogenetics at diagnosis
The researchers also want to assess the value of having a stem cell transplant using cells from a donor for people taking part in this trial.
Who can enter
You may be able to join this trial if all of the following apply. You
- Have acute myeloid leukaemia (AML) or myelodysplastic syndrome (MDS) that is considered to be high risk because more than 10% of your bone marrow is made up of immature cells called blasts
- Are over the age of 60
- Have satisfactory results of blood tests showing how well your liver and kidneys are working
- 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 6 months afterwards 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 a type of AML called acute promyelocytic leukaemia (APL)
- Have already had chemotherapy for AML apart from a low dose of hydroxycarbamide (or a similar drug)
- Have chronic myeloid leukaemia (CML) that is turning into acute myeloid leukaemia (this stage of CML is called the blast phase)
- Have any other type of cancer apart from basal cell skin cancer
- Are pregnant or breastfeeding
- Are known to have HIV
You cannot join the part of the trial looking at adding the drug quizartinib if you have certain heart problems or take medication that could affect your heart (the trial team can advise you about this). Or if you are going to join the group of people having vosaroxin and decitabine.
This is a phase 2/3 trial. The researchers need 1,600 people to take part. At different points in the trial, the people taking part are put into groups by computer. Neither they, nor their doctors can decide which group they are in. This is called randomisation.
You have chemotherapy in cycles of treatment. Each cycle is made up of the time when you have the drugs and a period of rest for your body to recover. Your medical team will explain more about how and when you have the chemotherapy drugs at each point in the trial.
For people with low risk cytogenetics
In the first part of the trial, researchers are looking at chemotherapy and Mylotarg.
As induction treatment, people with known poor risk
- A cycle of DA chemotherapy and 1 dose of Mylotarg
- A cycle of DA chemotherapy and 2 doses of Mylotarg
You have the Mylotarg through a drip into a vein on the first day of your chemotherapy cycle. If you have 2 doses of Mylotarg, you have the 2nd one on the 4th day.
After this cycle of chemotherapy, the researchers will look at samples of your blood and
If you are in complete remission and don’t have any MRD, you will have 1 more cycle of DA. If you are still in complete remission with no signs of MRD after this, you will be randomised to have either
- 1 more cycle of DA chemotherapy (3 cycles in total)
- 1 cycle of IDAC chemotherapy
If you aren’t in complete remission after the 1st cycle of chemotherapy, or you have MRD (or the doctors don’t know whether or not you have MRD), you are randomised to have 1 of the following
- 2 more cycles of DA
- 2 more cycles of DA and another chemotherapy drug called cladribine
- 1 cycle of chemotherapy called FLAG-Ida, followed by 1 cycle of chemotherapy called mini FLAG-Ida (people over 70 will have mini FLAG-Ida for both cycles)
FLAG-Ida is made up of the drugs
Mini FLAG-Ida is made up of the same drugs, but some are at lower doses.
After the 1st cycle of chemotherapy, everybody taking part may also be randomised to have either quizartinib or no drug.
If you are in the quizartinib group, you take it as tablets once a day for 2 weeks after each cycle of chemotherapy. When you have finished chemotherapy, you take quizartinib tablets for another 28 days. Some people will continue to take it for up to a year. Your doctor will explain if this applies to you.
For people with known high risk cytogenetics
You have at least 2 and up to 5 cycles of treatment with the chemotherapy drugs:
The number of cycles of treatment you have depends on your needs.
You have both drugs as a drip into a vein. It takes up to 10 minutes to have vosaroxin and about an hour to have decitabine. You stay in hospital for about 5 days during each cycle treatment.
As well as the treatments described above, you may be able to have a stem cell transplant if a suitable donor is available.
The trial team will ask you to fill out questionnaires before you start treatment, after 3 months, 6 months and a year. The questionnaires will ask about side effects and how you’ve been feeling. This is called a quality of life study.
You have up to 3 cycles of chemotherapy lasting 4 to 6 weeks each. You are likely to be in hospital for between 3 and 5 weeks in each cycle of treatment.
Your doctor or specialist nurse will explain how and when you have the different drugs in this trial and how often you will need to go to hospital for tests and treatment.
The most common side effects of the chemotherapy drugs include
- Feeling or being sick
- Sore mouth
- Hair loss
- A drop in blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness
The most common side effects of Mylotarg include
- Shivering when you have the drug
- Changes to your liver
Quizartinib is a new drug, there may be side effects we don’t know about yet. The possible side effects include:
- High temperature
- Lung infections
- Tiredness (fatigue)
- Feeling sick
- A drop in the number of red blood cells (anaemia)
- Loss of appetite
If you have quizartinib, you have regular heart traces (
Your doctor or specialist nurse will talk to you about all the possible side effects of the different drugs before you start treatment.
How to join a clinical trial
Professor Nigel Russell
Cancer Research UK
Experimental Cancer Medicine Centre (ECMC)
National Cancer Research Institute (NCRI) AML Working Party
This is Cancer Research UK number CRUK/12/043.