RecruitingPHASE3INTERVENTIONAL

Revumenib in Combination With Azacitidine + Venetoclax in Patients NPM1-mutated or KMT2A-rearranged AML

This study is testing a new medicine called revumenib for adults with a type of blood cancer called Acute Myeloid Leukemia (AML). This is specifically for patients whose AML has certain genetic changes (called NPM1 or KMT2A mutations) and who aren't able to have very strong chemotherapy. Currently, these patients are often treated with a combination of two drugs, azacitidine and venetoclax. The study wants to find out if adding revumenib to this standard treatment can make it more effective. Revumenib works by blocking a specific molecule that these particular types of AML cells need to grow. The study will compare patients receiving the new drug alongside standard treatment to those receiving a dummy drug (placebo) alongside standard treatment, all while keeping patients and doctors unaware of which group they are in.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Stichting Hemato-Oncologie voor Volwassenen Nederland

Enrolment target

415

Start

31 Mar 2025

Estimated completion

10 Jul 2031

Acute Myeloid Leukemia, Adult

What is this study about?

This study is about finding better treatments for a type of blood cancer called Acute Myeloid Leukemia, or AML. Specifically, it focuses on patients who have AML with certain changes in their cells' genetic material, known as NPM1 or KMT2A mutations. These mutations mean the cancer cells rely on a specific molecule called 'menin' to grow. For patients with these types of AML who also can't have very strong, intensive chemotherapy, current treatment usually involves a mix of two drugs: azacitidine and venetoclax. While this treatment has helped many, it often doesn't cure the cancer, and it can come back.

That's where a new drug called revumenib comes in. Revumenib is designed to block that 'menin' molecule that the NPM1 or KMT2A-mutated AML cells depend on. By blocking menin, revumenib aims to stop these particular cancer cells from growing. The main goal of this study is to see if adding revumenib to the current standard treatment (azacitidine and venetoclax) can make the treatment more effective and improve outcomes for patients with these specific types of AML.

This is a 'Phase 3' study, which means it's a larger study designed to confirm if the new treatment is better than the existing one. It's also 'randomised' (patients are put into groups by chance), 'double-blind' (neither the patients nor their doctors know which treatment they are getting), and 'placebo-controlled' (one group gets the new drug, the other gets a dummy drug alongside the standard treatment). This helps make sure the results are fair and accurate. After treatment, patients will be followed for up to four years to see how long they live and how they are doing.

Key takeaways

This study is for adults with a specific type of AML called NPM1-mutated or KMT2A-rearranged AML.
It's for people who cannot have intensive chemotherapy.
The study tests if adding a new drug, revumenib, to standard treatment helps more.
You might receive revumenib or a dummy drug (placebo), plus your usual treatment.
Participation includes regular check-ups and follow-up for up to four years.
You can leave the study at any time.

Who may be eligible?

This study is looking for around 415 adult patients with newly diagnosed Acute Myeloid Leukemia (AML). To be considered, your AML must have specific genetic changes — either an NPM1 mutation or a KMT2A rearrangement. These are crucial details that your medical team will confirm.

Another very important point is that this study is for patients who, for various reasons, cannot have the very strong 'intensive chemotherapy' that is sometimes used for AML. This might be because you are aged 75 or older, or if you are between 18 and 74 but have other health conditions that make intensive chemotherapy too risky. These conditions could include certain heart or lung problems, kidney issues, or other health concerns that your doctor believes would make intensive chemotherapy unsafe for you.

This study is for adults aged 18 and over, with no upper age limit. Your doctor will also need to believe that you are likely to live for at least another 12 weeks to participate. They will check your medical history and test results carefully to make sure you meet all the criteria for joining the study.

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 an adult aged 18 or older?
Has your doctor confirmed your AML has an NPM1 mutation or KMT2A rearrangement?
Has your doctor said that you are not suitable for intensive chemotherapy?
Do you have a general health status that your doctor believes is compatible with joining the study?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be assigned by chance to one of two groups: either you'll receive the new drug, revumenib, plus the standard combination of azacitidine and venetoclax, or you'll receive a dummy drug (placebo) plus the standard combination. Neither you nor your doctor will know which group you are in. You will continue to receive treatment until your AML gets worse, you experience side effects that make continuing difficult, or unfortunately, if you pass away.

Throughout the treatment period, you will have regular visits to the clinic for assessments, blood tests, and to monitor your health and any side effects. Your medicine will be given as prescribed by the study doctors. After you stop treatment, whether it's due to your AML progressing or for other reasons, the study team will continue to follow your health for up to four years. This follow-up involves occasional check-ups to record how you are doing, how long you live, and to gather important information for the study.

Potential risks and benefits

Taking part in any medical study carries both potential benefits and risks. You might benefit from receiving a new drug, revumenib, which could potentially work better than the existing treatment for your specific type of AML. However, it's also possible that revumenib may not help you, or it could cause side effects that are not yet fully known. As with any drug, there's always a chance of unpleasant reactions. Your doctors will carefully monitor you for any side effects and manage them as best as possible. You are absolutely free to leave the study at any time, for any reason, without it affecting your ongoing medical care.

Locations (67)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

DE-Berlin-CAMPUSBENFRANKLIN
Verified postcode
Berlin, Germany· Not yet recruiting
DE-Berlin-CAMPUSVIRCHOW
Verified postcode
Berlin, Germany· Not yet recruiting
DE-Berlin-VIVANTESNEUKOLLN
Verified postcode
Berlin, Germany· Not yet recruiting
DE-Bochum-RUB
Verified postcode
Bochum, Germany· Not yet recruiting
DE-Bonn-UNIBONN
Verified postcode
Bonn, Germany· Not yet recruiting
DE-Braunschweig-KLINIKUMBRAUNSCHWEIG
Verified postcode
Braunschweig, Germany· Not yet recruiting
DE-Bremen-KBM
Verified postcode
Bremen, Germany· Not yet recruiting
DE-Darmstadt-KLINIKUMDARMSTADT
Verified postcode
Darmstadt, Germany· Not yet recruiting
DE-Essen-KEM
Verified postcode
Essen, Germany· Not yet recruiting
DE-Flensburg-MALTESER
Verified postcode
Flensburg, Germany· Not yet recruiting
DE-Frankfurt-KLINIKUMFRANKFURT
Verified postcode
Frankfurt, Germany· Not yet recruiting
DE-Freiburg-UNIKLINIKFREIBURG
Verified postcode
Freiburg im Breisgau, Germany· Not yet recruiting

Common questions

What is AML?

AML stands for Acute Myeloid Leukemia, which is a type of cancer that affects the blood and bone marrow, the soft tissue inside your bones where blood cells are made.

What does NPM1-mutated or KMT2A-rearranged mean?

These are specific genetic changes found in the AML cells of some patients. They tell us more about the type of AML you have and how it might respond to certain treatments.

What is a 'placebo'?

A placebo is a dummy drug that looks like the real treatment but has no active medicine. It helps researchers fairly compare how well the new drug works.

Why can't I have intensive chemotherapy?

For some people, strong chemotherapy can be too hard on the body because of age or other health conditions. This study is specifically for those patients.

How long will I be in the study?

You'll continue treatment as long as it's helping and you're not experiencing bad side effects. After treatment, your health will be checked for up to four years.

How to find out more

Gerwin Huls, MD

HOVON@erasmusmc.nl +31-(0)107041560 Official trial record

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