AuthorisedTherapeutic exploratory (Phase II)Interventional

A Phase II, Open-Label, multicentre, study investigating tolerability and efficacy of Gilteritinib in combination with fludarabine, cytarabine and idarubicin (FLAI) as induction therapy of newly diagnosed non-M3 FLT3-positive acute myeloid leukemia.GIMEMA AML2924

This clinical trial is for adults recently diagnosed with a specific type of acute myeloid leukaemia (AML) that has a genetic change called FLT3. Researchers are testing a new drug called Xospata (gilteritinib) alongside standard chemotherapy. The main goal is to see how many patients achieve remission after the first one or two courses of treatment, meaning their leukaemia is no longer detectable. They will also look at how long patients stay in remission, their overall health, and any side effects from the new combination treatment. This is a Phase II study, meaning it's exploring the effectiveness and safety of a promising new treatment approach in a group of patients.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Fondazione Gimema Franco Mandelli Onlus

Enrolment target

Start

04 Jul 2025

newly diagnosed non-M3 FLT3-positive acute myeloid leukemia

What is this study about?

This study is about a type of blood cancer called acute myeloid leukaemia (AML). This particular study focuses on AML that has just been diagnosed and has a specific genetic marker, or change, called FLT3. This marker helps doctors understand more about the cancer and choose the best treatments.

The study combines a new drug called Xospata with a standard chemotherapy treatment. The main aim is to find out how well this new combination works in getting rid of the leukaemia. Doctors will be looking at how many patients go into remission after one or two rounds of treatment. Remission means there are very few, or no, leukaemia cells left in the body.

They will also be checking for other important things, like how long the remission lasts, how the treatment affects patients' overall health, and if there are any side effects. Researchers will also look at how many patients can then go on to have a stem cell transplant, which can be an important next step for some people with AML. They will also collect information on side effects and how patients' quality of life is during treatment. This is a Phase II study, which means it's an important step in seeing if this new treatment could be a good option for future patients.

Key takeaways

This study is for adults with newly diagnosed 'FLT3-positive' AML.
It combines a new drug (Xospata) with standard chemotherapy.
The main goal is to see how many patients achieve remission and how safe the combination is.
It's a Phase II study, exploring the treatment's effectiveness.
Participation involves regular hospital visits and follow-up.

Who may be eligible?

To join this study, you must be an adult, aged 18 or older, who has recently been diagnosed with a specific type of acute myeloid leukaemia (AML). This AML must have a particular genetic change called FLT3, and it must not be a subtype known as M3 AML.

Both men and women can take part. The research team will do some tests to make sure that the treatment is suitable for your specific health situation.

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 acute myeloid leukaemia (AML)?
Do your AML cells have the 'FLT3' genetic marker?
Is your AML not classified as 'M3 type'?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll receive the study medication, Xospata, along with standard chemotherapy drugs. You'll have regular hospital visits for treatments, blood tests, and check-ups to see how you're responding and to monitor for any side effects. This will involve several treatment cycles. After your treatment ends, you'll continue to have follow-up appointments, which may include visits to the hospital or phone calls, to monitor your long-term health and wellbeing. The total duration of your participation will vary depending on your treatment response and follow-up schedule.

Potential risks and benefits

Potential benefits of joining this study include access to a new treatment combination that might be more effective than standard treatments alone for your type of leukaemia. However, there are also potential risks, as all medical treatments can have side effects. These will be explained in detail by the study team, and you will be carefully monitored throughout the trial. It's important to remember that participating is entirely voluntary, and you have the right to withdraw from the study at any time without affecting your 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
Italy

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 'FLT3-positive' mean?

'FLT3-positive' means that the leukaemia cells have a specific genetic change that doctors can identify. This helps them understand the type of leukaemia.

What is Xospata?

Xospata is the brand name for a drug called gilteritinib. It's a targeted therapy that works by blocking the FLT3 gene change.

What is 'remission'?

Remission means that tests show very few or no signs of leukaemia in your body. It doesn't always mean the cancer is cured, but it's a very positive sign.

Will I still get standard chemotherapy?

Yes, in this study, the new drug Xospata is given in combination with standard chemotherapy drugs.