RecruitingPhase IIIInterventional

Phase III study of revumenib in combination with intensive chemotherapy in newly diagnosed NPM1-mutated AML

This study is investigating if a new medication called revumenib, when combined with standard chemotherapy, can improve treatment for people recently diagnosed with a specific type of blood cancer called acute myeloid leukemia (AML) that has an NPM1 gene change. AML is an aggressive cancer, and while current treatments can help, many patients experience their cancer returning. Revumenib is a tablet designed to target a specific process involved in AML. This trial is hoping to find a more effective first treatment. Participants will be randomly given either revumenib or a dummy pill alongside chemotherapy, and neither they nor their doctors will know which they are receiving. The study will monitor their progress and any side effects. It's for people aged 12 and over who are suitable for intensive chemotherapy.

At a glance

Status

Recruiting

Phase

Phase III

Sponsor

Syndax Pharmaceuticals, Inc.

Enrolment target

468

Start

16 Mar 2026

Estimated completion

30 Nov 2027

Newly diagnosed acute myeloid leukemia (AML) with an NPM1 mutation

What is this study about?

This research study is looking into a new way to treat a serious blood cancer called Acute Myeloid Leukemia (AML). Specifically, it's for people who have been recently diagnosed with AML that also has a particular genetic change, or 'mutation', called NPM1. AML is an aggressive cancer, and while current treatments, often involving strong chemotherapy, can help get the cancer into remission (a period where the cancer is under control), it sadly often comes back.

The main aim of this study is to see if adding a new drug called revumenib to the usual chemotherapy treatments can make a difference. Revumenib is a tablet that works by targeting a specific pathway in the body that is involved in how this type of AML develops. It's currently an 'investigational' drug, which means it's not yet approved for use outside of research studies like this one. If the study shows that revumenib is helpful, it could lead to a better first-time treatment option for people with this specific type of AML.

This is an important piece of research because it could potentially lead to improved outcomes for patients with NPM1-mutated AML. By carefully studying how revumenib works alongside standard treatments, scientists hope to find more effective ways to fight this challenging disease.

Key takeaways

New study for newly diagnosed AML with a specific gene change (NPM1 mutation).
Compares standard chemotherapy plus new drug (revumenib) versus standard chemotherapy plus dummy pill.
Revumenib is an experimental oral medication (a pill).
Participants will be aged 12 years and older.
You won't know if you're getting the new drug or the dummy pill (placebo).
Involves regular tests like blood tests, bone marrow checks, and scans.
Aims to find a more effective first treatment for this type of AML.

Who may be eligible?

To be considered for this study, you must have been recently diagnosed with AML and have the specific NPM1 gene change. You also need to be well enough to receive strong chemotherapy treatments. Your doctor will carefully check your overall health, including how well your liver, kidneys, and heart are working, to make sure it's safe for you to take part. This study is open to people aged 12 and older.

There are also some reasons why you might not be able to join. For example, if you have a different type of AML called acute promyelocytic leukemia, or if your cancer has spread to your brain or spinal cord. Certain heart conditions, serious stomach issues that might stop you from absorbing medication, or other serious health problems could also prevent you from participating. You also cannot be pregnant or breastfeeding, or have certain active infections like hepatitis B, hepatitis C, or uncontrolled HIV.

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.

Have I been recently diagnosed with AML?
Has my doctor confirmed I have the NPM1 gene change?
Am I generally well enough for strong chemotherapy?
Am I 12 years old or older?
Do I have any serious heart, liver, or kidney problems?
Am I able to swallow tablets?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be randomly assigned to one of two groups: one group will receive the new drug, revumenib, along with standard chemotherapy, and the other group will receive a 'dummy pill' (placebo) along with standard chemotherapy. This is a "blinded" study, meaning neither you nor your doctors will know which group you are in. This helps to make the results as fair and accurate as possible.

Throughout the study, you will have regular check-ups. These will involve blood tests to see how you are responding to treatment and to check for any side effects. You will also have bone marrow biopsies (where a small sample of bone marrow is taken, usually from your hip bone) and imaging scans, like X-rays or CT scans. These tests help the doctors monitor your condition and understand how the treatment is working. The study is expected to run from October 2025 to January 2031, with your individual participation duration being discussed with your study doctor.

Potential risks and benefits

There is no guarantee that taking part in this study will directly improve your health, and it's possible your condition could get worse. However, by participating, you will be contributing to vital research that could help future patients with AML. Like all medical treatments, there are potential risks and possible side effects associated with the study drugs and tests. You will receive a detailed information sheet that explains all the potential side effects and burdens. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (10)

The Royal Marsden NHS Foundation Trust
City only
London, England
University Hospitals Birmingham NHS Foundation Trust
City only
Birmingham, England
Aberdeen Royal Infirmary
Approximate
Aberdeen, Scotland
Imperial College Healthcare NHS Trust
City only
London, England
Oxford University Hospitals NHS Foundation Trust
City only
Oxford, England
University Hospital Southampton NHS Foundation Trust
City only
Southampton, England
Belfast Health and Social Care Trust
City only
Belfast, Northern Ireland
United Lincolnshire Teaching Hospitals NHS Trust
City only
Lincoln, England
Beatson West of Scotland Cancer Centre
City only
Glasgow, Scotland
University Hospitals of Derby and Burton NHS Foundation Trust
City only
Derby, England

Common questions

What exactly is NPM1-mutated AML?

AML is a blood cancer. NPM1-mutated means your cancer has a specific change in its genes. This study is focused on this particular type of AML.

What is revumenib?

Revumenib is a new experimental drug, given as a pill, that aims to block a specific process involved in AML. It's not yet approved for general use but is being tested in this study.

Will I definitely get the new drug?

No, you will be randomly assigned to either receive revumenib or a dummy pill (placebo) alongside your standard chemotherapy. Neither you nor your doctor will know which one you get.

How long will the study last for me?

The study itself runs from October 2025 to January 2031. Your individual participation length will be discussed by the study team, but it will involve ongoing treatment and monitoring.

Who is paying for this study?

The study is funded by Syndax Pharmaceuticals, Inc., a company based in the USA, who are also running the study.

How to find out more

Syndax Pharmaceuticals

clinicaltrials@syndax.com +1 (0)781 419 1400 Official trial record

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