A trial looking at new drugs in combination with cytarabine for acute myeloid leukaemia (AML LI-1)

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Acute leukaemia

Acute myeloid leukaemia (AML)

Blood cancers

Leukaemia

Myelodysplastic syndrome (MDS)

Status:

Closed

Phase:

Phase 2/3

This trial is looking at new drugs in combination with cytarabine (Ara C) for acute myeloid leukaemia and high risk myelodysplastic syndrome (MDS).

More about this trial

Doctors often treat acute myeloid leukaemia (AML) and high risk MDS with intensive chemotherapy to try and get rid of it. But some people can’t or don’t want to have intensive chemotherapy. For these people doctors may use the chemotherapy drug cytarabine at a lower dose than in intensive chemotherapy. They know cytarabine works but are always looking for new treatments.

In this trial some people will have cytarabine and others will have cytarabine in combination with newer drugs. The researchers will compare the groups to find out which is best for treating people with AML and high risk MDS.

Who can enter

You may be able to enter this trial if you

Have acute myeloid leukaemia (AML) or myelodysplastic syndrome (MDS)
Have satisfactory blood test results
Are willing to use reliable contraception if your partner could become pregnant
Are 60 years of age or older – some younger patients may be able to take part

You cannot enter this trial if you

Have acute promyelocytic leukaemia (APL)
Have already had chemotherapy to treat AML – apart form hydroxycarbamide or similar treatment to control your white blood cells
Have chronic myeloid leukaemia (CML) that may be in the process of turning into AML (blast transformation)
Are being treated for another cancer
Have had a heart attack or heart pain (angina) that is not controlled by medication or a stroke in the last 6 months
Are HIV positive
Are pregnant or breastfeeding

You can't have BCT-100 if you have had another drug that works in a similar way. But you may still be able to take part in this trial and have one of the other drugs.

Trial design

This is a phase 2/3 trial. It will recruit 1,000 people.

It is a randomised trial. You are put into a treatment group by a computer. Neither you nor your doctor can choose which group you are in. The groups are:

Cytarabine and AC220
Sapacitabine
Cytarabine (which is the standard treatment)
Cytarabine and vosaroxin
Vosaroxin
Cytarabine and ganetespib
Cytarabine and tosedostat
Cytarabine and selinexor
Cytarabine and lenalidomide
Cytarabine and BCT-100

Please note: People are no longer been put into the following treatment groups:

Vosaroxin
Sapacitabine
Cytarabine and vosaroxin
Cytarabine and ganetespib
Cytarabine and tosedostat
Cytarabine and selinexor
Cytarabine and AC220
Cytarabine and lenalidomide

Not all of the treatment groups will be available at every centre, all of the time. Your doctor will tell you which ones are available to you.

The treatments repeat every 4 to 6 weeks, depending on the treatment you are having and how your AML responds to treatment. Each 4 to 6 week period is called a cycle of treatment.

You have cytarabine as an injection under the skin. You have it twice a day for 10 days every 4 to 6 weeks. You may be able to have this at home. You can talk to your doctor about this.

You have BCT-100 as a drip into a vein (intravenous). You have it once a week for 4 weeks at the start of each cycle of treatemnt.

You have at least 4 cycles of treatment. You may continue having treatment as long as it is helping you.

You fill in a questionnaire before starting treatment and at 3, 6 and 12 months. It will ask about how you have been and any side effects you have. This is called a quality of life study.

The researchers will also ask your permission to store blood and bone marrow samples. They will take the blood samples when you have your routine samples taken. And the bone marrow samples from when you had your bone marrow test done. These samples will be used in future to find out more about AML. You don’t have to agree to these if you don’t want to. You can still take part in the main trial.

The researchers may also ask for additional blood samples if you are having BCT-100. This is to find out more about the how the drug is working.