A trial looking at venetoclax for acute myeloid leukaemia

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

Status:

Closed

Phase:

Phase 3

This trial is looking at venetoclax with low dose chemotherapy for acute myeloid leukaemia (AML). It is for people with AML who haven’t had treatment and aren’t able to have intensive chemotherapy.

More about this trial

The main treatment to get rid of AML is intensive chemotherapy. This is a combination of:

  • cytarabine
  • an anthracycline Open a glossary item chemotherapy drug such as daunorubicin

If you aren’t fit enough to have intensive chemotherapy your doctor might give you low dose cytarabine.

Venetoclax is a targeted drug. It works by blocking a protein found in AML cells. This causes the AML cells to die. From previous research we know that venetoclax might help people with AML.

The team want to find out if combining venetoclax with low dose cytarabine can help people who can’t have intensive chemotherapy.

In this trial, 2 out of every 3 people taking part have venetoclax and low dose cytarabine. And 1 out of every 3 people taking part have a dummy drug (placebo Open a glossary item) and low dose cytarabine.

The main aims of this trial are to find out:

  • how well venetoclax with low dose cytarabine works for people with AML who can’t have intensive chemotherapy
  • what the side effects are
  • how this combination affects your quality of life Open a glossary item

Who can enter

The following bullet points list the entry conditions for this trial. Talk to your doctor or the trial team if you are unsure about any of these. They will be able to advise you.

You may be able to join this trial if all of the following apply:

  • you have acute myeloid leukaemia and can’t have intensive induction chemotherapy
  • your kidneys work well enough, your doctor will do a test for this
  • you have satisfactory blood test results
  • you are willing to use reliable contraception during the trial and for at least 6 months afterwards if there is any chance you or your partner could become pregnant
  • you are at least 18 years old

One of the following must also apply. You:

  • are 75 years old or older, are able to look after yourself and are up and about for more than half the day (performance status 0, 1 or 2)

Or

  • are between 18 years old and 74 years old, are able to look after yourself and are up and about for more than half the day OR need help to look after yourself and are in bed or a chair for more than half the day (performance status 0, 1, 2 or 3)

You cannot join this trial if any of these apply.

Cancer related
You:

  • have already had treatment for AML apart from hydroxyurea (hydroxycarbamide). You might be able to join the trial if you have had treatment for a myelodysplastic syndrome Open a glossary item (MDS) unless it included cytarabine 
  • had a myeloproliferative neoplasm Open a glossary item (MPN) before AML
  • have acute promyelocytic leukaemia (APL)
  • have AML that has spread to your brain or spinal cord
  • have had another cancer apart from carcinoma in situ Open a glossary item of the cervix or breast, non melanoma skin cancer Open a glossary item or any other cancer that hadn’t spread and had been treated with the aim to cure

Medical conditions
You:

  • have HIV
  • have hepatitis B or hepatitis C, you might be able to join if you are a carrier of hepatitis and it isn’t active or it is controlled by medication
  • are taking medication that affects the CYP enzymes
  • have moderate to severe heart problems that affects your daily activities
  • have an ongoing breathing problem that means you need to have continuous oxygen
  • have or have had serious problems with your kidneys
  • have a problem with your digestive system Open a glossary item that affects how your body absorbs food and medications
  • have an infection that affects your whole body and needs treatment
  • have a high number of white blood cells Open a glossary item in your blood, you might be able to join the trial if you are able to have hydroxyurea or a procedure called leukapheresis to bring the number of white cells in your blood down
  • have any other medical or mental health problem that your doctor thinks could affect you taking part in the trial

Other
You:

  • have eaten grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or star fruit within 3 days of starting treatment in the trial
  • are pregnant or breastfeeding

Trial design

This is an international phase 3 trial. The researchers need 175 people worldwide to join including up to 6 people from the UK.

This is a randomised trial. You are put into 1 of 2 treatment groups and neither you nor your doctor can choose which group you are in.

  • venetoclax and low dose cytarabine
  • dummy drug (placebo Open a glossary item) and low dose cytarabine

You have twice as much chance of going into the venetoclax group than the dummy drug group.

Trial Diagram

Neither you nor your doctor will know which group you are in. This is a double blind trial.

Venetoclax and the dummy drug are tablets. You take them within 30 minutes of finishing breakfast or your first meal of the day.

You take increasing doses of venetoclax, or the dummy drug, until you are taking the maximum dose. This can take about 5 days. You stay in hospital during this time so the nurses can monitor you.

You have the low dose cytarabine as an injection under the skin. You have it for 10 days every 4 weeks.

You usually come into the hospital each day to have the cytarabine. Or your doctor might be able to arrange for you to have it at home or at your local GP surgery, if possible. They will talk to you about this.

You continue having treatment for as long as it is helping and the side effects aren’t too bad.

You have a diary to fill in. You write down when you had your tablets and how many. You bring this with to every appointment.

Samples
The team will take extra blood samples, tissue samples and bone marrow samples.

The team will also ask if they can take blood samples to look at the genes Open a glossary item inside the cells. This is a genetic sub study. You don’t have to agree to this if you don’t want to, you can still take part in the main trial.

They will use these samples to find:

  • why some people’s leukaemia responds better to treatment than others
  • more about AML and how to treat it
  • what happens to venetoclax in the body

Quality of life
You fill in a few questionnaires:

  • at the start of treatment
  • every 8 weeks while on treatment
  • at the end of treatment

The questions ask about how you are feeling and any side effects you might have. This is a quality of life study

Hospital visits

You see the doctor to have some tests before joining the trial. These include:

  • a physical examination
  • blood tests
  • urine test
  • breathing test (lung function test Open a glossary item)
  • heart scan (MUGA Open a glossary item or ECHO Open a glossary item)
  • heart trace (ECG Open a glossary item)
  • bone marrow test

You are in hospital for at least 5 days at the start of treatment. When you leave hospital, you see the doctor or nurse at least once a week for 3 weeks. This is to have blood tests to see how you are.

During treatment you see the doctor every 4 weeks for blood tests and a physical examination. You have a bone marrow test at 4 weeks and then every 12 weeks.

At the end of treatment you see the doctor for the same tests you had at the start. You see them a month afterwards and then every 2 months.

Side effects

You are monitored closely for any side effects that might happen. The common side effects of venetoclax might include:

  • infection of the nose and throat causing a runny nose and, or a cough
  • a drop of white blood cells in the blood. You might have a high temperature (fever) as well, this is called febrile neutropenia
  • diarrhoea or constipation
  • tiredness
  • feeling or being sick
  • a lung infection (pneumonia)
  • urine infection
  • a high amount of phosphorous, uric acid, potassium, calcium and creatinine in the blood

When you first start treatment you might have tumour lysis syndrome. This is caused by the breakdown of the cells of the leukaemia. Your doctor might ask you to drink plenty of water before starting treatment. They might also give you some medication to take that will help your body get rid of this cell breakdown.

Signs of tumour lysis syndrome include:

  • high temperature (fever)
  • chills
  • feeling or being sick
  • diarrhoea
  • confusion
  • shortness of breath
  • irregular heartbeat
  • unusual tiredness
  • muscle pain
  • sore joints
  • fits (seizures)

You must tell your nurse or doctor straight away if you have any of these signs. You are in hospital for at least 5 days when start treatment so the nurses and doctor can monitor you.

We have information about the side effects of cytarabine.

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.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Caroline Alvares

Supported by

AbbVie

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

15218

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Wendy took part in a new trial studying the possible side effect of hearing loss

A picture of Wendy

"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”

Last reviewed:

Rate this page:

No votes yet
Thank you!
We've recently made some changes to the site, tell us what you think