CompletedPhase IIInterventional

A trial comparing the effectiveness and safety of venetoclax to standard chemotherapy in acute myeloid leukaemia patients

Acute myeloid leukaemia (AML) is an aggressive blood cancer. The standard treatment for fit patients is intensive chemotherapy. While this can cure the condition, it causes severe side effects such as dangerous infections, hair loss, and long hospital stays. This can be especially tough on older adults. This trial tests a newer targeted drug called venetoclax, taken as a daily tablet, combined with low-dose chemotherapy injections. Early studies show that venetoclax works very well in AML, particularly for people whose leukaemia cells carry a specific gene change called NPM1. Researchers want to see if venetoclax is as effective as standard intensive chemotherapy. Patients aged 55 or older will be randomly put into one of two groups to receive either the venetoclax treatment or standard chemotherapy. Doctors will closely monitor everyone's health and blood counts over two years.

At a glance

Status

Completed

Phase

Phase II

Sponsor

University of Birmingham

Enrolment target

156

Start

01 Mar 2021

Estimated completion

01 Jun 2026

Acute myeloid leukaemia

What is this study about?

Acute myeloid leukaemia (AML) is a fast-growing cancer of the white blood cells that affects over 3,000 people in the UK each year. Doctors usually treat fit patients with intensive chemotherapy to destroy the leukaemia cells and cure the disease. However, intensive chemotherapy is very demanding on the body. It wipes out healthy blood cells, leaves people open to serious infections, and often requires several weeks of staying in the hospital.

Doctors are searching for kinder treatments that can kill leukaemia cells without causing such heavy side effects. A newer drug called venetoclax has shown very promising results in earlier trials, especially in people with a specific genetic subtype called NPM1-mutated AML. For many patients, venetoclax managed to clear the cancer just as effectively as intensive chemotherapy.

The VICTOR trial was designed to directly compare venetoclax (taken alongside gentle, low-dose chemotherapy) against standard intensive chemotherapy. If venetoclax proves to be just as effective, it could become a new standard treatment. Because venetoclax comes as a tablet you can take at home, it could allow patients to spend far less time in hospital and enjoy a much better quality of life during their treatment.

Key takeaways

The VICTOR trial compares a newer targeted drug (venetoclax) with standard intensive chemotherapy for AML.
It is open to people aged 55 and over who have newly diagnosed AML with an NPM1 gene change.
Venetoclax is taken as an oral tablet at home, which may mean fewer hospital stays and milder side effects.
Participants are randomly assigned to one of the two treatment groups and followed for two years.
Your health and response to treatment are monitored closely throughout, with the option to switch therapies if needed.

Who may be eligible?

You may be able to join this study if you are 55 or older and have newly diagnosed acute myeloid leukaemia (AML). Your leukaemia cells must test positive for a protein called CD33 and have a specific genetic change known as an NPM1 mutation (without another change called FLT3-ITD). Your doctor must also feel you are physically fit enough to have standard intensive chemotherapy.

You will not be able to take part if you have already had chemotherapy for AML in the past (though a short course of a gentle medicine called hydroxycarbamide to manage your blood counts is allowed). People with another active cancer that needs treatment, or those with uncontrolled viral infections such as HIV or hepatitis, cannot take part. Pregnant or breastfeeding women are also unable to join.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

I am 55 years of age or older.
I have been newly diagnosed with acute myeloid leukaemia (AML).
My leukaemia has been confirmed to have the NPM1 mutation.
My doctor considers me fit enough for intensive chemotherapy.
I have not had previous chemotherapy for AML (except hydroxycarbamide to manage blood counts).
I do not have another active cancer requiring treatment.

Answer every question to see your result.

What does participation involve?

If you take part, a computer will randomly place you into one of two treatment groups. You have an equal chance of being in either group. Group one receives the trial treatment: daily venetoclax tablets taken at home, alongside low-dose cytarabine injections, given in 28-day cycles for up to 12 cycles. If this goes well, you may take venetoclax on its own for another year. Group two receives standard intensive chemotherapy given into a vein in the hospital for up to 4 cycles.

Whichever group you are in, your medical team will track your progress very carefully using regular blood tests and bone marrow checks. If your leukaemia does not respond well enough, your doctor will switch your treatment immediately or discuss other options like a stem cell transplant. Total follow-up for the study lasts for two years.

Potential risks and benefits

A potential benefit is that venetoclax may be gentler than standard chemotherapy, allowing you to take tablets at home with fewer harsh side effects and shorter hospital stays. However, because this is a trial, doctors do not yet know if venetoclax is as effective at curing the leukaemia as standard treatment. You may also experience side effects from the study medicines, such as low blood counts or tummy upset, as well as minor bruising or discomfort from regular blood and bone marrow tests. Taking part is completely voluntary, and you are free to leave the trial at any point without it affecting your standard NHS care.

Locations (44)

John Radcliffe Hopsital
City only
Oxford, England
Central Manchester University Hospitals NHS Foundation Trust
City only
Manchester, England
Victoria Hospital
Approximate
Blackpool, England
Guy’s Hospital
Approximate
London, England
Queens Hospital
Approximate
Romford, England
The Royal London Hospital
Unverified
London, England
University College London Hospital
Unverified
London, England
Queen Elizabeth Hospital Birmingham
Unverified
Birmingham, England
St. Mary's Hospital
Unverified
London, England
The Royal Marsden Hospital
Unverified
London, England
Belfast City Hospital
Unverified
Belfast, Northern Ireland
Bristol Royal Infirmary
Unverified
Bristol, England

Common questions

Can I choose which treatment I get?

No. A computer randomly decides which group you join. This ensures the study results are fair and reliable.

Can I take venetoclax at home?

Yes. Venetoclax is taken as a tablet by mouth, which means you can take it at home rather than staying in hospital for long drips.

What happens if the trial treatment does not work for me?

Your care team will monitor your blood and bone marrow closely. If the cancer does not improve as hoped, your doctor will stop the trial medicine and switch you to another proven treatment.

Does it cost anything to join the trial?

No. The trial is run through the NHS and funded by Cancer Research UK. All study treatments and extra tests are provided at no cost to you.

Can I leave the study if I change my mind?

Yes, you can leave the study at any time. You do not need to give a reason, and your regular NHS doctors will continue to give you the best available care.

How to find out more

Catherine Thomas

victor@trials.bham.ac.uk +44 (0)121 371 7861 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.