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Study of Gilteritinib, Venetoclax, and Azacitidine for Patients with Newly Diagnosed Acute Myeloid Leukemia and FLT3 Mutations Not Eligible for Intensive Treatment

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a type of blood cancer called Acute Myeloid Leukemia (AML), specifically in patients who have a genetic change known as FLT3 mutations. The study is designed for patients who have been newly diagnosed with AML and are not eligible for intensive treatment. The trial will explore the use of three medications: Gilteritinib (also known by its code name ASP2215), Venetoclax (also known as ABT-199), and Azacitidine. Gilteritinib is a type of medication called a tyrosine kinase inhibitor, which helps block certain proteins that promote cancer cell growth. Venetoclax is a cytostatic, meaning it helps stop cancer cells from dividing, and Azacitidine is a hypomethylating agent, which can help restore normal function to cancer cells.

The purpose of the study is to find the best dose of Gilteritinib when used in combination with Venetoclax and Azacitidine. The study will last for about 12 months, during which participants will receive these medications in a specific sequence. The trial will monitor how well patients tolerate the treatment and how effective it is in managing their leukemia. Participants will take Gilteritinib and Venetoclax in the form of tablets, while Azacitidine will be given as an injection under the skin. The study will also include a placebo group to compare the effects of the medications.

Throughout the study, researchers will assess the ratio of the dose delivered to the dose planned, which will help determine both the safety and effectiveness of the treatment. The trial aims to improve understanding of how these medications can be used together to treat AML with FLT3 mutations, providing valuable information for future treatment options. Participants will be closely monitored for any side effects and the overall response to the treatment. The study is expected to conclude by the end of 2028.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial treatment phase
The treatment begins with a combination of medications designed to address acute myeloid leukemia with FLT3 mutations.
The first medication, azacitidine, is administered subcutaneously. This means it is injected under the skin. The specific dosage and frequency will be determined by the healthcare provider based on individual needs.
The second medication, venetoclax, is taken orally in the form of film-coated tablets. The dosage and frequency are also tailored to the patient's condition and response to treatment.
Step 2
Introduction of gilteritinib
After the initial phase with azacitidine and venetoclax, gilteritinib is introduced. This medication is also taken orally as film-coated tablets.
The goal is to find the most effective and tolerable dose when combined with the other medications. The healthcare team will monitor the patient's response closely.
Step 3
Monitoring and adjustment
Throughout the treatment, the healthcare team will regularly assess the patient's response to the medication combination.
Adjustments to dosages may be made to optimize effectiveness and minimize side effects. This process is crucial to ensure the best possible outcome.
Step 4
Evaluation of treatment efficacy
The primary measure of success is the ratio of the dose delivered to the dose planned over a 12-month period. This reflects both how well the treatment is working and how well it is tolerated.
Secondary evaluations include remission status, duration of response, and overall survival rates. These assessments help determine the long-term benefits of the treatment.
Step 5
Completion of treatment
The estimated end date for the trial is October 31, 2028. By this time, all data will be collected and analyzed to understand the full impact of the treatment regimen.
Patients will continue to be monitored for any long-term effects or benefits of the treatment.

Who can join the trial?

13 criteria

Must provide a signed informed consent, which means you agree to participate in the study after understanding all the details.
Must have been newly diagnosed with **acute myeloid leukemia (AML)**, a type of blood cancer, according to specific medical criteria, with a certain level of cancer cells in the bone marrow.
Must have a specific genetic change called **FLT3 mutation** at the initial diagnosis, which is determined by a local laboratory test.
Must be at least 18 years old.
Must be unable to undergo standard intensive treatment due to age (75 years or older) or if aged 18 to 74, must have certain health conditions such as:
- A performance status of 2 or 3, which means a certain level of physical activity limitation.
- A history of heart problems like heart failure or stable chest pain.
- Reduced lung function.
- Reduced kidney function.
- Moderate liver problems.
- Any other health condition that makes intensive chemotherapy unsuitable, as judged by a doctor.
- Must have had one cycle of a specific pre-treatment with drugs called VEN and AZA, and have results from a bone marrow test on day 21 of this treatment.
- Male participants must agree to avoid unprotected sex and not donate sperm from the time of signing the consent until 4 months after the last dose of the study drug.
- Women who can become pregnant must have a negative pregnancy test within 3 days before the first dose of the study drug.

Who cannot join the trial?

6 criteria

Patients who have received intensive treatment for their condition.
Patients without the specific genetic change called FLT3 mutation.
Patients who are eligible for intensive treatment.
Patients who are not diagnosed with acute myeloid leukemia, a type of blood cancer.
Patients who are not adults, as the study is for adult participants only.
Patients who are not part of the vulnerable population, which includes groups that may need special protection.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Goethe University Frankfurt
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Frankfurt, Germany
Klinikum der Universitaet Muenchen AöR
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Munich, Germany
Technische Universitaet Dresden
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Dresden, Germany

Trial locations

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Investigated drugs

Gilteritinib
is a medication used in this trial to treat patients with acute myeloid leukemia (AML) who have specific genetic mutations known as FLT3 mutations. It works by targeting and inhibiting the activity of the FLT3 protein, which is often overactive in these patients, helping to slow down or stop the growth of cancer cells.
Venetoclax
is another medication used in this trial. It helps treat AML by targeting a protein called BCL-2, which helps cancer cells survive. By inhibiting this protein, venetoclax can promote the death of cancer cells, making it an effective part of the treatment regimen.
Azacitidine
is a medication that is part of the standard treatment for AML. It works by interfering with the growth of cancer cells, helping to slow down their proliferation. Azacitidine is often used to improve blood cell counts and reduce the symptoms of leukemia.

What is already known about the treatment

Gilteritinib
This medication is administered orally and is currently being studied in combination with other drugs for treating acute myeloid leukemia (AML) with FLT3 mutations. It is not yet fully established in medical practice but is under investigation in clinical trials. Gilteritinib is primarily indicated for patients with AML who are not eligible for intensive treatment. It works by inhibiting the FLT3 receptor, which is involved in the growth and survival of cancer cells. Gilteritinib is classified as a tyrosine kinase inhibitor.
Venetoclax
This medication is taken orally and is being evaluated in combination with other treatments for acute myeloid leukemia. It is already used in some medical settings but is being further studied for its effectiveness in specific patient groups. Venetoclax is indicated for patients with AML who cannot undergo intensive treatment. It functions by blocking the BCL-2 protein, which helps cancer cells survive, leading to their death. Venetoclax is classified as a BCL-2 inhibitor.
Azacitidine
Administered via injection, this medication is part of the standard treatment being tested in combination with other drugs for acute myeloid leukemia. It is well-established in medical literature and practice for certain blood disorders. Azacitidine is used for patients with AML who are not suitable for intensive treatment. It works by incorporating into DNA and RNA, disrupting the growth of cancer cells. Azacitidine is classified as a nucleoside metabolic inhibitor.

Investigated diseases

Acute Myeloid Leukemia – Acute myeloid leukemia is a type of cancer that starts in the blood-forming cells of the bone marrow. It progresses rapidly, leading to the accumulation of abnormal white blood cells that interfere with the production of normal blood cells. This can result in symptoms such as fatigue, frequent infections, and easy bruising or bleeding. The disease can spread to other parts of the body, including the lymph nodes, liver, and spleen. It is more common in older adults and can vary in its specific genetic mutations, which may influence its behavior and response to treatment.