Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Randomized, Open-label, Controlled, Phase 2 Study of Pevonedistat, Venetoclax, and Azacitidine Versus Venetoclax Plus Azacitidine in Adults With Newly Diagnosed Acute Myeloid Leukemia Who Are Unfit for Intensive Chemotherapy Triple Combination of Pevonedistat and Venetoclax Plus Azacitidine in Adults With Acute Myeloid Leukemia Who Are Unfit for Intensive Chemotherapy

Doctors are studying new ways to treat Acute Myeloid Leukemia (AML) in adults who aren't well enough for very intense chemotherapy. This research involves comparing two different treatment approaches. One group of patients will receive a combination of three medicines: pevonedistat, venetoclax (also known as Venclyxto), and azacitidine (also known as Vidaza or Azacitidin STADA). The other group will receive a combination of two medicines: venetoclax and azacitidine. The main goal is to see how long patients live without their disease getting worse or coming back, or without them passing away. Researchers will also look at how long people live overall, whether fewer people die within 30 or 60 days of starting treatment, and how well the treatment shrinks or clears the cancer.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Takeda Development Center Americas Inc.

Enrolment target

Start

09 Oct 2024

Acute Myeloid Leukemia

What is this study about?

This study is a clinical trial looking at new treatments for a type of blood cancer called Acute Myeloid Leukemia (AML). AML affects your white blood cells and can be very serious. This particular study is for adults who have recently been diagnosed with AML and are considered too unwell or frail to undergo very strong chemotherapy (intensive chemotherapy).

The trial wants to find out if adding a new drug called pevonedistat to a standard two-drug treatment (venetoclax plus azacitidine) works better than the standard two-drug treatment alone. Venetoclax and azacitidine are already used to treat AML. By comparing these two approaches, doctors hope to find a more effective way to help people who can't tolerate the usual intensive chemotherapy.

The main thing the doctors will be watching for is called "event-free survival." This means they'll be tracking how long people live without significant problems like the cancer not going away, coming back, or passing away. They will also look at how long people live overall and how well the treatments reduce or get rid of the cancer.

Key takeaways

This study explores new treatments for AML in adults who cannot tolerate strong chemotherapy.
It compares a three-drug combination against a two-drug combination to see which is more effective.
The main focus is on how long people live without their disease worsening or returning.
Participation involves regular hospital visits for treatment, monitoring, and health checks.
You have the right to leave the study at any point without impacting your care.

Who may be eligible?

This study is designed for adults aged 18 and over. It's specifically for people who have been diagnosed with a type of blood cancer called Acute Myeloid Leukemia (AML) for the first time.

Crucially, participants must be considered by their doctors to be 'unfit' for strong (intensive) chemotherapy. This usually means that their general health or other medical conditions make the side effects of very powerful chemotherapy too risky for them.

Both men and women can take part in this study, as long as they meet the other specific health requirements set by the research team. There will be other detailed health checks to make sure the treatment is safe and appropriate for everyone involved.

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 Leukemia (AML)?
Has your doctor said you are not able to have strong (intensive) chemotherapy?
Are you willing to have regular hospital visits, blood tests, and examinations?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive either the three-drug combination (pevonedistat, venetoclax, and azacitidine) or the two-drug combination (venetoclax and azacitidine). Which treatment you get is decided by chance, like flipping a coin, so neither you nor your doctor can choose. All drugs will be carefully managed by the study team.

You would have regular visits to the hospital or clinic to receive your medication, have blood tests, and undergo examinations. These visits are important for monitoring your health, checking how the treatment is working, and looking for any side effects. The study will also track blood levels of pevonedistat to ensure the right dose.

The total duration of your participation could vary. You'll receive treatment for a period determined by your health and how the cancer responds. Even after treatment stops, the study team will likely follow up with you for some time to understand the long-term effects of the treatment and how you are doing overall.

Potential risks and benefits

Taking part in any clinical trial has potential benefits and risks. A potential benefit of this study is that you might receive a new treatment combination that could be more effective for your AML, especially if you can't have intensive chemotherapy. However, there's no guarantee the treatment will work for everyone, and it might not work better than existing treatments. There are also potential risks involved, mainly from the side effects of the drugs. These could include feeling unwell, changes in your blood counts, infections, or other reactions. The study team will explain all known side effects. Remember, you have the right 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
Italy
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Unverified
Poland

Common questions

What is Acute Myeloid Leukemia (AML)?

AML is a type of cancer that starts in the bone marrow, the soft inside part of your bones where new blood cells are made. It causes too many abnormal white blood cells to be produced.

What does 'unfit for intensive chemotherapy' mean?

It means a doctor believes that very strong chemotherapy, which has many side effects, would be too harmful or risky for your general health, often due to age or other medical conditions.

What are venetoclax and azacitidine?

These are medicines already used to treat AML. Venetoclax (Venclyxto) targets specific proteins in cancer cells, while azacitidine (Vidaza) works by changing how cells grow and divide.

Will I know which treatment I'm getting?

In this specific study, you will not know if you are receiving the two-drug or three-drug combination. This is a common way to conduct studies to ensure fair comparison without bias.

How long does the study last?

The time you spend on treatment varies depending on how you respond. The study team will continue to monitor your health and progress for a period even after treatment stops.