Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3 Multicenter, Open-label, Randomized Study of ASP2215 (Gilteritinib), Combination of ASP2215 Plus Azacitidine and Azacitidine Alone in the Treatment of Newly Diagnosed Acute Myeloid Leukemia with FLT3 Mutation in Patients Not Eligible for Intensive Induction Chemotherapy

This research is a study for adults newly diagnosed with a specific type of blood cancer called Acute Myeloid Leukaemia (AML) that has a gene change called a FLT3 mutation. It's for people who aren't able to have very strong initial chemotherapy. The study will compare different treatments: a drug called gilteritinib on its own, gilteritinib combined with another drug called azacitidine, and azacitidine on its own. We want to find out if these new approaches help people live longer. We'll also look at how well the treatments work to control the cancer, how people feel, and any side effects. This is a "Phase 3" study, meaning it's a bigger trial to confirm if a new treatment is better than existing ones.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Astellas Pharma Global Development Inc.

Enrolment target

Start

12 Jun 2024

Newly Diagnosed Acute Myeloid Leukemia with FLT3 Mutation in Patients Not Eligible for Intensive Induction Chemotherapy

What is this study about?

This study is looking into better ways to treat a specific type of blood cancer called Acute Myeloid Leukaemia (AML). AML affects white blood cells, and the type we're focusing on has a particular change in a gene called FLT3. This study is for adults who have been newly diagnosed with this type of AML and, importantly, are not well enough to receive very strong initial chemotherapy treatments.

The main goal of this research is to see if a drug called gilteritinib, either by itself or when given along with another drug called azacitidine, can help people live longer compared to just receiving azacitidine alone. Azacitidine is a standard treatment for this condition. By finding out which treatment works best, doctors can make more informed decisions about how to care for patients in the future.

We'll also be looking at several other important things. This includes how effectively the treatments reduce the cancer cells, how long people stay in remission (meaning the cancer is under control), how they feel in terms of tiredness, and what side effects they might experience. This helps us get a full picture of the benefits and challenges of each treatment option.

Key takeaways

This study is for adults with newly diagnosed AML with a FLT3 gene mutation.
It's specifically for those who cannot easily receive strong initial chemotherapy.
We are comparing gilteritinib (a new drug) with standard treatment (azacitidine).
The main aim is to see if these new treatments help people live longer.
Participation involves regular check-ups and monitoring for effects and side effects.

Who may be eligible?

To join this study, you must be an adult, aged 18 or older, and have recently been diagnosed with a type of blood cancer called Acute Myeloid Leukaemia (AML). Importantly, your AML needs to have a specific gene change called a FLT3 mutation. We'll check for this with a special test.

Another key requirement is that you must not be a suitable candidate for very strong chemotherapy treatments, often called 'intensive induction chemotherapy.' This might be because of your age, other health conditions, or how well your body can cope with aggressive treatments. Your doctor will help determine if this applies to you.

Both men and women can take part in this study, as long as they meet all the other criteria. The research team will review your medical history to make sure the study is a safe and appropriate option 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 recently been diagnosed with Acute Myeloid Leukaemia (AML)?
Has your doctor told you that your AML has a 'FLT3 mutation'?
Has your doctor said you aren't suitable for very strong first chemotherapy treatments?
Are you able to attend regular appointments and tests?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be randomly assigned to one of three treatment groups: receiving gilteritinib on its own, gilteritinib combined with azacitidine, or azacitidine on its own. This is like flipping a coin, so neither you nor your doctor can choose which group you join. You'll receive your assigned study medication and have regular check-ups with the study team.

These check-ups will involve things like blood tests, physical examinations, and possibly heart tests (ECGs) to monitor your health and how the treatment is working. We'll also ask you questions about how you're feeling, including about your energy levels or tiredness. The treatment will continue for as long as it's helping you and you're not experiencing severe side effects. After your main treatment, there will be follow-up appointments to track your long-term health.

The total time you're involved could vary, but it includes the treatment period and follow-up. The study team will explain the full schedule of visits and tests so you know exactly what to expect before you agree to participate.

Potential risks and benefits

Potential benefits of joining this study include receiving a new investigational treatment (gilteritinib) that might be more effective than standard treatment for your specific type of AML. You'll also receive close medical attention and monitoring throughout the study. However, like all medications, the study drugs may have side effects, which could range from mild to serious. These will be fully explained by the study team. There's also no guarantee that the treatment will work for you. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

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

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Unverified
Germany
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Unverified
Belgium

Common questions

What is AML with FLT3 mutation?

It's a type of blood cancer (Acute Myeloid Leukaemia) where the white blood cells don't grow properly, and it has a specific gene change called a FLT3 mutation.

What does 'not eligible for intensive chemotherapy' mean?

It means your doctor believes you're not well enough to safely receive very strong initial chemotherapy treatments, often due to age or other health issues.

What is gilteritinib?

Gilteritinib is a study drug designed to target the FLT3 gene change in AML cells, hoping to stop them from growing.

Will I definitely get gilteritinib?

You will be randomly assigned to one of three groups, so you might receive gilteritinib alone, gilteritinib with another drug, or the usual treatment only.

How long will I be in the study?

The time you spend in the study will depend on how you respond to treatment and how long you continue with medication, plus a follow-up period. The team will provide more details.