RecruitingPhase IIIInterventional

A study of bleximenib, venetoclax and azacitidine for treatment of participants with newly diagnosed acute myeloid leukemia (cAMeLot-2)

This research trial, called cAMeLot-2, is investigating a new medication named bleximenib for people who have recently been diagnosed with a type of blood cancer called acute myeloid leukaemia (AML). This study is for patients whose AML has specific genetic changes (KMT2Ar or NPM1m) and who aren't suitable for tough chemotherapy. Bleximenib works by targeting certain proteins that help leukaemia cells grow. Researchers want to compare how well bleximenib, when given with two other drugs (Venetoclax and Azacitidine), works against a placebo combined with those same two drugs. The goal is to find out if this new combination can improve outcomes for people with this aggressive cancer.

At a glance

Status

Recruiting

Phase

Phase III

Sponsor

Janssen-Cilag International NV

Enrolment target

600

Start

14 Apr 2025

Estimated completion

19 Sep 2027

Acute myeloid leukemia

What is this study about?

Acute myeloid leukaemia (AML) is a serious blood cancer where too many immature white blood cells build up in the bone marrow, affecting the body's ability to fight infections. Currently, treatment options can be limited, and many patients can't have standard, strong chemotherapy because it's too harsh for them. This means new and kinder treatments are really needed to help people with AML.

This study is looking at a new drug called bleximenib. Bleximenib works in a special way: it targets and blocks the interaction between two proteins, KMT2A and menin, which are often involved in AML. For patients whose AML has certain genetic changes (called KMT2A rearrangements or NPM1 mutations), blocking these proteins can help kill the leukaemia cells and stop the disease from getting worse. Researchers want to see if adding bleximenib to two existing AML drugs, Venetoclax and Azacitidine, works better than just using Venetoclax and Azacitidine with a dummy pill (placebo).

This is a Phase III study, which means it’s one of the final steps before a new drug might be approved for wider use. By comparing the new combination with existing treatment, the study aims to understand if bleximenib can offer a better treatment option for patients with newly diagnosed AML who have these specific genetic changes and are unable to have intensive chemotherapy. This research is important for potentially improving future care for people living with this condição.

Key takeaways

This study is for newly diagnosed AML with specific genetic changes.
It tests a new drug, bleximenib, combined with existing treatments.
Participation requires careful monitoring and regular medical checks.
There are potential benefits, but also risks and side effects to consider.
You won't know if you're getting the new drug or a placebo combined with standard treatment.
You can withdraw from the study at any time without penalty.

Who may be eligible?

This study is for adults, aged 18 or older, who have recently been diagnosed with acute myeloid leukaemia (AML). To join, your AML must have specific genetic changes called KMT2A rearrangements (KMT2Ar) or NPM1 mutations (NPM1m). This is a key requirement, as the new drug is designed to work for these specific types of AML.

Crucially, you must also be considered unsuitable for intensive chemotherapy. This might be due to your overall health or other medical conditions. You'll also need to have healthy liver and kidney function to ensure your body can handle the study treatments. Both men and women can participate, but if you are a woman, you must not be pregnant or breastfeeding and must agree to use effective birth control during and for six months after the study. Men must also agree to use effective birth control during and for six months after the study.

There are also some reasons why you couldn't join. For example, if you have a different type of leukaemia called acute promyelocytic leukaemia (APL), if the leukaemia has spread to your brain or spinal cord, or if you've recently had an organ transplant. Certain heart conditions, active hepatitis, recent vaccinations, or known severe allergies to the study drugs would also prevent you from participating.

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 old or older?
Have you recently been diagnosed with AML that has KMT2A or NPM1 genetic changes?
Are you considered unsuitable for intensive chemotherapy by your doctor?
Do you have generally healthy liver and kidney function?
Are you able and willing to use effective birth control if needed?

Answer every question to see your result.

What does participation involve?

If you decide to join the study, it will be split into three main parts. First, there's a 'screening' phase, which can last up to 28 days. During this time, you'll have tests to make sure you meet all the requirements to take part.

Next is the 'treatment' phase. You'll be assigned by chance (like flipping a coin) to one of two groups: one group will receive the new drug bleximenib along with Venetoclax and Azacitidine. The other group will receive a dummy pill (placebo) along with Venetoclax and Azacitidine. You and your doctors won't know which group you're in, to keep the results fair. You'll continue treatment for as long as it's helping you, or until any side effects become too difficult, or if the study team decides it's no longer suitable for you.

Finally, there's a 'follow-up' phase. During this time, the study team will keep in touch to check on your general health and how you're doing. Throughout the study, you'll have regular blood and urine tests, as well as physical examinations. The team will carefully keep track of any side effects you might experience. The entire study is expected to last about four and a half years.

Potential risks and benefits

It's important to understand that while this study is trying a new treatment, there's no guarantee that participating will directly benefit you. However, based on how bleximenib works, scientists hope that adding it to existing treatments might improve outcomes for people with AML, but this isn't certain yet. Even if you receive the placebo, you will still get the standard treatment of Venetoclax and Azacitidine. You might also find some benefit from the regular check-ups and close monitoring of your health during the study, and your participation will certainly help future patients with AML by contributing to medical knowledge. On the other hand, all medications and procedures have possible side effects, and those in this study range from mild to severe, and could even be life-threatening. Potential side effects of bleximenib could include immune reactions (like 'differentiation syndrome'), issues arising from many leukaemia cells dying quickly ('tumour lysis syndrome'), low blood counts, infections, or changes to your heart rhythm. There might also be effects on fertility and other risks not yet known. The study team will monitor you closely, explain all known risks in detail before you agree to participate, and provide care for any side effects. You are free to withdraw from the study at any time without affecting your future medical care.

Locations (9)

Western General Hospital
City only
Lothian, United Kingdom
Addenbrookes
Approximate
Cambridge, United Kingdom
Derriford Hospital
Approximate
Plymouth, United Kingdom
Kent & Canterbury Hospital
Approximate
Canterbury, United Kingdom
Royal Sussex County Hospital
Approximate
Brighton, United Kingdom
Colchester
Approximate
Colchester, United Kingdom
Clatterbridge Cancer Centre
Approximate
Liverpool, United Kingdom
Guys Hospital
City only
London, United Kingdom
Worthing Hospital
Approximate
Worthing, United Kingdom

Common questions

What is Acute Myeloid Leukaemia (AML)?

AML is a type of blood cancer where abnormal white blood cells develop in your bone marrow, which then stops your body from making healthy blood cells.

What does 'newly diagnosed' mean in this study?

It means the study is for people who have only recently found out they have AML and haven't had previous strong treatments for it yet.

What are KMT2Ar and NPM1m?

These are specific genetic changes found in some people's AML cells. The new drug, bleximenib, is designed to work particularly well for these types of AML.

What is a 'placebo'?

A placebo is a dummy pill or treatment that looks the same as the active drug but contains no active medicine. It helps researchers compare the effects of the active drug accurately.

Will I have to pay to be in this study?

No, there are no costs for you to participate. The study treatment and tests are paid for, and you may receive reasonable reimbursement for travel and parking expenses.

How to find out more

Ana Moreno

JanssenUKRegistryQueries@its.jnj.com - Official trial record

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