AuthorisedTherapeutic exploratory (Phase II)Interventional

An open-label, phase 2 study, evaluating the efficacy of treatment with Gilteritinib in measurable residual disease eradication in patients with FLT3-ITD mutated acute myeloid leukemia

This important study is testing a new treatment approach for adults with a type of blood cancer called acute myeloid leukaemia (AML) that has a specific gene change (FLT3-ITD). We are using a medicine called Xospata to see if it can get rid of even the smallest traces of cancer cells, which doctors call 'measurable residual disease' (MRD) negativity. We will check this after two cycles of treatment. This study is ‘open-label,’ meaning everyone involved will know they are receiving Xospata. It's a Phase 2 study, which means we are exploring how well the treatment works and gathering more information about its safety. The results will help us understand if this treatment could be a better way to fight this specific type of leukaemia.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Hellenic Society Of Hematology

Enrolment target

Start

20 Nov 2025

Acute myeloid leukaemia Acute myeloid leukaemia

What is this study about?

This study is focused on a specific type of blood cancer called acute myeloid leukaemia (AML). This particular AML has a genetic change called FLT3-ITD. We are looking at a medicine called Xospata (gilteritinib) to see how effective it is in treating this condition.

The main goal is to find out if Xospata can completely get rid of what doctors call 'measurable residual disease' (MRD). This refers to very tiny amounts of cancer cells that might still be in the body, even if they can't be seen with standard tests. Achieving MRD negativity is important because it can mean a better outlook for patients.

We will also be looking at other things, like how long the medicine works before the cancer might start to resist it. We'll examine changes in the cancer cells themselves and try to understand what features in a person's body or the cancer cells might predict how well they will respond to Xospata. This will help doctors understand who might benefit most from this treatment in the future.

Key takeaways

This study is for adults with a specific type of blood cancer (AML with FLT3-ITD).
It aims to see if the drug Xospata can completely clear tiny cancer cells (MRD).
Researchers will monitor how patients respond and look for reasons why some might respond better than others.
The findings could help improve future treatments for this condition.

Who may be eligible?

To be considered for this study, you need to be an adult, aged 18 years or older. There is no upper age limit for taking part.

This study includes both men and women. The specific type of blood cancer you have is also very important – it must be acute myeloid leukaemia (AML) with a specific genetic change called FLT3-ITD.

Your doctor will check other important health details to make sure this study is right for you and that you meet all necessary criteria for safety. It's important to discuss all your health conditions with your doctor.

Quick self-check

Are you 18 years old or older?
Have you been diagnosed with acute myeloid leukaemia (AML)?
Does your AML have the FLT3-ITD genetic change?
Are you able to take tablet medication?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will receive the study medicine, Xospata, in tablet form (40 mg). You will take this medicine for at least two cycles. During these cycles, doctors will take samples (like blood samples or bone marrow samples) to check for those tiny traces of cancer cells (MRD) to see if they have disappeared. They will also monitor you closely for any side effects and how your body is reacting to the treatment.

There will be regular visits to the clinic for assessments and check-ups. The doctors will monitor how the cancer cells might change over time and look at your genetic information and immune system. The exact number of visits, tests, and total length of your participation will be explained in detail by the study team.

Potential risks and benefits

Taking part in this study could potentially offer a benefit by providing access to a new treatment, Xospata, which might help clear up the remaining cancer cells. However, as with any medicine, there are potential risks, including side effects from the drug, which your doctor will discuss with you. There might also be discomfort or minor risks from procedures like blood tests. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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Greece

Common questions

What is FLT3-ITD?

FLT3-ITD is a specific genetic change found in some types of acute myeloid leukaemia (AML) that the study is focusing on.

What does 'measurable residual disease' (MRD) mean?

MRD refers to small numbers of cancer cells that can be found in the body, even after treatment, which are too few to be seen with standard tests.

What is Xospata?

Xospata is the name of the medicine being tested in this study for treating AML with the FLT3-ITD genetic change.

What does 'Phase 2 study' mean?

A Phase 2 study means researchers are exploring how well a new treatment works and continue to gather information on its safety for a specific condition.

Will I know if I'm getting the study drug?

Yes, this is an 'open-label' study, which means everyone involved, including you and your doctors, will know you are receiving Xospata.