AuthorisedPhase I and Phase II (Integrated)- OtherInterventional

ABT-199 (Venetoclax) and Purine analogues as Novel Oral Drug Combination for Treatment of Relapsed/Refractory Acute Myeloid Leukemia (ApoAML)

This study is for adults diagnosed with Acute Myeloid Leukaemia (AML), a type of blood cancer, where the disease has either returned after treatment or didn't get better with previous treatments. Scientists are testing a new combination of two drugs, venetoclax and mercaptopurine, to see if they can effectively fight the cancer. This is a multi-stage study, starting with Phase I to check for safety and the right dose, then moving to Phase II to see how well the treatment works in a larger group of people. The main goal is to see if the cancer goes away or significantly improves, and to understand any side effects.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Antwerp University Hospital

Enrolment target

Start

13 Feb 2024

Acute myeloid leukemia

What is this study about?

This study is investigating a new way to treat Acute Myeloid Leukaemia (AML) in adults. AML is a type of cancer that affects the blood and bone marrow. Sometimes, even after treatment, AML can come back (this is called 'relapsed') or it might not get better with the usual treatments (called 'refractory'). This can be a very challenging situation.

In this study, researchers are looking at two specific medications: venetoclax and mercaptopurine. Both are taken by mouth. Venetoclax works by targeting a protein in cancer cells that helps them survive, while mercaptopurine is a type of chemotherapy. The idea is that using these two drugs together might be more effective than using them separately for people with AML that has returned or hasn't responded to other treatments.

The study is split into two parts: Phase I and Phase II. In Phase I, the main focus is to make sure the drug combination is safe and to find the best dose. Once a safe and effective dose is identified, the study moves to Phase II. In this phase, more people will receive the treatment to see how well it works at treating the AML and to gather more information about any side effects.

Key takeaways

New combination treatment for AML.
For adults with relapsed or refractory AML.
Combines venetoclax and mercaptopurine (oral drugs).
Aims to assess safety and effectiveness.
Study has two parts: Phase I (safety/dose) and Phase II (effectiveness).

Who may be eligible?

This study is looking for adults aged 18 and over, with no upper age limit, who have been diagnosed with Acute Myeloid Leukaemia (AML).

You might be able to join if your AML has come back after previous treatment, or if your AML didn't respond well to the treatments you've already had. Both men and women are welcome to take part.

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.

I am 18 years old or older.
I have been diagnosed with Acute Myeloid Leukaemia (AML).
My AML has either come back after treatment OR it didn't get better with previous treatments.
I am able to take medications by mouth.

Answer every question to see your result.

What does participation involve?

The study involves taking two medications by mouth: venetoclax and mercaptopurine. You would have regular check-ups, which include blood tests and possibly bone marrow biopsies, to see how the treatment is working and to monitor for any side effects. The frequency of visits and tests would likely be more intensive at the beginning of the study. The total duration of your participation would depend on how your AML responds to the treatment and your overall health, but you would be regularly monitored throughout.

Potential risks and benefits

Taking part in this study could potentially offer a new treatment option for your Acute Myeloid Leukaemia if other treatments haven't worked or if your cancer has returned. However, there are potential risks, as with any medication, including side effects from venetoclax and mercaptopurine. These will be carefully explained to you by the study team. You always have the right to withdraw from the study at any time, for any reason, without it affecting your usual 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
Belgium

Common questions

What is Acute Myeloid Leukaemia (AML)?

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

What does 'relapsed/refractory' mean?

Relapsed means your AML has come back after treatment. Refractory means your AML didn't get better with previous treatments.

What are venetoclax and mercaptopurine?

They are two different medications. Venetoclax helps target cancer cells, and mercaptopurine is a type of chemotherapy, both taken by mouth.

What is a 'clinical response'?

This is how doctors measure how well the treatment is working. It means looking for signs that the cancer is going away or significantly improving.

Will I get one of the study drugs or a placebo?

In this specific study, all participants will receive the combination of venetoclax and mercaptopurine. There is no placebo group.