Not yet recruitingPhase IIInterventional

Comparing a new combination of medicines to the usual intensive chemotherapy treatment given to participants who have been recently diagnosed with acute myeloid leukaemia

This research study is for adults diagnosed with a type of blood cancer called Acute Myeloid Leukaemia (AML). We want to find out if a new combination of four medicines (tagraxofusp, venetoclax, cytarabine, and cladribine) is safer and works better than the usual strong chemotherapy treatments. The main goal is to help more patients become well enough to have a bone marrow transplant, which can cure AML. Participants will either receive the new combination or one of the standard treatments. The study will involve about 220 people across the UK and will follow them for up to seven years to see how they respond.

At a glance

Status

Not yet recruiting

Phase

Phase II

Sponsor

Didact Foundation

Enrolment target

222

Start

30 Jun 2026

Estimated completion

01 Jun 2029

Newly diagnosed Acute Myeloid Leukaemia

What is this study about?

This study is designed for adults who have recently been diagnosed with a specific type of blood cancer called Acute Myeloid Leukaemia, often shortened to AML. AML affects the bone marrow, which is where new blood cells are made. The main purpose of this research is to compare a new combination of four medicines (tagraxofusp, venetoclax, cytarabine, and cladribine) against the standard intensive chemotherapy treatments currently given to patients. We want to see if this new combination is a safer and more effective way to treat AML.

The ultimate aim of this study is to increase the number of patients who can successfully receive a bone marrow transplant. A transplant can be a very effective treatment for AML, but patients need to be in good health to go through with it. By finding better initial treatments, we hope more people will reach this important stage. The study will involve two main parts, with the new treatment being given to all participants in the first part, and then compared to standard care in the second, larger part.

We expect about 220 people from around the UK to take part in this study. Participants in the second part will be randomly assigned to either receive the new four-drug combination or one of the usual strong chemotherapy regimens. Patients will receive treatment for at least two cycles and will be followed closely for up to seven years to understand the long-term effects and benefits.

Key takeaways

Compares a new four-drug treatment to standard chemotherapy for AML.
Aims to help more patients qualify for a potentially curative bone marrow transplant.
Involves adults with newly diagnosed 'Adverse' or 'Intermediate' risk AML.
Participants will be closely monitored for up to 7 years.
Participation involves routine care plus some extra tests and questionnaires.

Who may be eligible?

This study is looking for adults who have recently been diagnosed with Acute Myeloid Leukaemia (AML).

You might be able to take part if you are 18 years or older and have a type of AML known as 'Adverse risk' AML. Or, if you are 50 years or older and have 'Intermediate risk' AML. You would also need to be considered fit enough by your doctor for strong chemotherapy treatments.

There are also some reasons why you might not be able to join, such as having certain other medical conditions, already having received specific treatments for AML (though some types of early treatment are allowed), or having certain types of genetic changes in your AML cells. Your doctor will carefully check all these details with you.

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.

Are you 18 years or older?
Have you been recently diagnosed with Acute Myeloid Leukaemia (AML)?
Is your AML classified as 'Adverse risk' (if 18+) or 'Intermediate risk' (if 50+)?
Has your doctor said you are well enough for strong chemotherapy treatments?
Do you not have certain other serious health conditions or specific genetic mutations in your AML cells?

Answer every question to see your result.

What does participation involve?

If you join this study, it's designed to fit as closely as possible with the usual care you would receive for AML, so it shouldn't add much extra burden. You would go through a screening period to check if the study is right for you, then receive study treatment, followed by a period after treatment ends, and then long-term follow-up for up to seven years.

During the study, you'll have blood tests and bone marrow samples taken, like you normally would. The main extra things for the study would be a repeat bone marrow and blood sample at the start for additional research, a pregnancy test for women who could become pregnant when treatment stops, and you'll be asked to fill out some questionnaires about your quality of life at about 4-6 different times. Your treatment will involve at least two cycles, and the total follow-up could last up to seven years.

Potential risks and benefits

We can't promise that you will directly benefit from taking part in this study, as not everyone responds to new treatments in the same way. However, the information gathered from this study is very important and could help us find better treatment options for future patients with AML. As with any medical treatment, there are potential risks. You will either receive standard chemotherapy treatments, which have known side effects like infections, tiredness, feeling sick, and hair loss, or the new four-drug combination. The new drugs also have potential side effects, including low blood counts, changes in mineral levels, possible fluid build-up, nausea, and fatigue. Your medical team will explain all the possible side effects specific to the treatment you might receive, and supportive medicines will be given to manage these. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

Churchill Hospital
Approximate
Oxford, England
University College London Hospital
City only
London, England
University Hospital Birmingham
Approximate
Birmingham, England
Bristol Haematology & Oncology Centre
Approximate
Bristol, England
King's College Hospital NHS Foundation Trust
City only
London, England

Common questions

What is Acute Myeloid Leukaemia (AML)?

AML is a type of cancer that starts in the bone marrow, affecting the blood-forming cells. It's a fast-growing cancer.

What is a 'Phase II' study?

A Phase II study means researchers are testing a new treatment combination in a larger group of people to see if it's safe and effective, after initial safety checks.

Will I definitely get the new treatment?

If you join the second part of the study, you'll be randomly assigned to either receive the new combination or one of the standard chemotherapy treatments, like drawing lots.

How long will I be in the study?

You'll receive at least two cycles of treatment, and then you'll be followed up for your health for up to seven years to see the long-term effects.

Who is paying for this study?

The study is funded by Stemline Therapeutics, a pharmaceutical company based in the Netherlands.

How to find out more

Susie Brown

susie.brown@act4patients.com +44 7933 181944 Official trial record

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