Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

MRD-guided treatment with pembrolizumab and azacitidine in NPM1mut AML patients with an imminent hematological relapse (PEMAZA)

This research study, called PEMAZA, is for adults with a type of blood cancer called acute myeloid leukaemia (AML). Specifically, it's for those who have a marker (NPM1 mutation) that suggests their cancer might return soon, even if they don't have symptoms yet. The study is investigating a new treatment approach that combines two medicines: pembrolizumab (also known as Keytruda) and azacitidine. Doctors want to see if this combination can prevent the cancer from coming back (relapsing) or delay its return. They'll be watching closely to see how many people stay well without their cancer returning for at least 24 weeks after starting treatment. This is a Phase II study, meaning it's an earlier-stage trial testing if the treatment is effective and safe.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Technische Universitaet Dresden

Enrolment target

Start

25 Oct 2024

Acute myeloid leukemia

What is this study about?

This study is about a type of blood cancer called acute myeloid leukaemia (AML). When someone has AML, their body makes abnormal white blood cells. Even after initial treatment, sometimes the cancer cells are still present in very small numbers, which can't always be seen with regular tests. Doctors call this 'minimal residual disease' (MRD). If this MRD is present, it means the AML might come back in the future.

The PEMAZA study is looking at adults with a specific type of AML (NPM1-mutated AML) who have MRD, suggesting their cancer could be about to return. The study aims to see if combining two existing medicines, called pembrolizumab (brand name Keytruda) and azacitidine, can help prevent the cancer from coming back or delay it. Pembrolizumab is an immunotherapy that helps your own immune system fight cancer, and azacitidine is a chemotherapy drug that works to stop cancer cells from growing. By giving these medicines together, researchers hope to get a better result than current treatments.

The main goal is to find out how many people remain free from their cancer returning, or from needing other AML treatment, or from passing away, after taking the drug combination for 24 weeks. This helps doctors understand if this combined treatment is effective at keeping the cancer under control for longer. The study is also looking at how long people live overall, and how the amount of MRD changes over time. This research is important because it could lead to better ways of preventing AML from coming back for people at high risk.

Key takeaways

This study explores a new treatment combination for AML.
It targets adult patients with a specific gene change (NPM1 mutation) at risk of relapse.
The treatment involves two drugs: pembrolizumab (Keytruda) and azacitidine.
The main goal is to see if the treatment can keep cancer away for at least 24 weeks.
This is a Phase II study, checking for effectiveness and safety.

Who may be eligible?

This study is open to adults who are 18 years old or older, with no upper age limit. Both men and women can take part.

You might be considered for this study if you have a specific type of acute myeloid leukaemia (AML) where a particular gene, called NPM1, has changed (mutated). This is important because the study focuses on people where there are still very small numbers of leukaemia cells present in their body, which means there's a risk of the cancer coming back soon.

Your doctors will need to do some special tests to confirm if you have this particular gene change and if you meet other specific medical requirements for the study. If you're interested, your specialist team can help determine if this study is right for 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 old or older?
Have you been diagnosed with Acute Myeloid Leukaemia (AML)?
Has your doctor told you that your AML has an 'NPM1 mutation'?
Are you considered at risk of your AML returning soon, based on special tests for minimal residual disease (MRD)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive treatment for up to 24 weeks. This involves getting the two study medications:

* **Azacitidine:** You would typically receive this medicine for 7 days in a row, once every four weeks. * **Pembrolizumab (Keytruda):** This medicine would be given as an infusion (into your vein) approximately every three weeks, for up to 8 doses in total over the 24 weeks.

Throughout the study, you would have regular appointments for check-ups, blood tests, and scans to monitor your health and how the treatment is working. Doctors will also check for any side effects. After the 24 weeks of active treatment, you would likely have follow-up visits to continue tracking your health and progress, though these might be less frequent. The total duration of your participation, including follow-up, would be explained by the study team.

Potential risks and benefits

Taking part in any clinical study has potential benefits and risks. A potential benefit of this study is that the combination of pembrolizumab and azacitidine might help prevent your AML from coming back or delay its return for a longer period than current standard treatments. However, this is not guaranteed, and the treatment might not work for everyone. Both medications can have side effects, which can range from mild to serious, and your doctor will discuss these with you in detail. You will be closely monitored for any side effects throughout the study. Remember, joining this study is completely voluntary, and you have the right to withdraw at any time, for any reason, without it affecting your future medical care.

Locations (1)

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

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Unverified
Germany

Common questions

What is AML?

AML stands for Acute Myeloid Leukaemia, which is a type of cancer that starts in the blood-forming cells of the bone marrow.

What does 'MRD-guided treatment' mean?

MRD-guided means your doctors are using highly sensitive tests to find tiny amounts of leukaemia cells (MRD) that can't be seen with normal tests. This helps them decide when to start treatment to try and stop the cancer from coming back.

What are pembrolizumab and azacitidine?

Pembrolizumab (Keytruda) is an immunotherapy drug that helps your immune system fight cancer. Azacitidine is a chemotherapy drug that aims to stop cancer cells from growing.

What does 'NPM1mut' mean?

NPM1mut means there's a change or 'mutation' in a gene called NPM1. This specific change is found in some types of AML and helps doctors identify people who might benefit from this particular study.

How long will the treatment last?

The active combination treatment with both medications will last for up to 24 weeks.