Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

With Ponatinib on the track for treatment-free-remission in chronic myeloid leukemia

This research is investigating a medicine called Iclusig (ponatinib) for people who have chronic myeloid leukaemia (CML). The main goal is to see if, after two years of taking Iclusig, people reach a very low level of the disease, known as MR4. This is a point where no disease can be detected using special tests, and it might mean they could potentially stop treatment later on. The study will also look at how patients feel, their quality of life, and any side effects. It aims to understand if a point can be reached where treatment might not be needed, offering hope for a 'treatment-free remission' journey.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Heidelberg University

Enrolment target

Start

22 Nov 2024

chronic myeloid leukemia in chronic phase

What is this study about?

This study is about a medicine called Iclusig (ponatinib), which is already approved for treating a type of blood cancer called chronic myeloid leukaemia (CML). People with CML have too many unusual white blood cells. Normally, treatment helps control the disease, but people usually have to take medicine for a long time.

The main idea behind this study is to see if taking Iclusig could help people with CML reach a stage where the disease is so well controlled that very sensitive tests can't find any trace of it. This is called 'MR4'. If someone reaches MR4 and stays there, it might open the door to them being able to stop their medicine in the future, which is known as 'treatment-free remission'. This could greatly improve their quality of life, as they wouldn't have to take daily medication anymore.

Researchers will closely follow people taking Iclusig for two years to see if they reach this MR4 target. They will also be looking at how safe the medicine is, what side effects people might experience, and how it affects their daily life and general well-being. By understanding these things better, doctors hope to find new ways to help people with CML live longer, healthier lives, potentially without needing continuous medication.

Key takeaways

Tests a medicine called Iclusig (ponatinib) for chronic myeloid leukaemia (CML).
Aims to see if patients can reach 'MR4' (no detectable disease) after two years.
MR4 could potentially allow patients to stop CML treatment in the future.
Will assess how safe the medicine is and its effects on quality of life.
Open to adults aged 18 and over with chronic phase CML.

Who may be eligible?

To join this study, you must be an adult, at least 18 years old, as there is no upper age limit. The study is open to both men and women.

The study is specifically for people who have chronic myeloid leukaemia (CML) and are currently in the 'chronic phase' of the disease. This means your CML is not advanced or in a blast crisis.

Your doctor will need to check other medical details to make sure this study is the right fit for you and that taking Iclusig would be safe. This might include reviewing your general health and any other medications you are taking.

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?
Do you have chronic myeloid leukaemia (CML)?
Is your CML currently in the 'chronic phase'?
Are you an adult who can take oral medication (tablets)?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be taking the study medicine, Iclusig, which comes as a 15 mg tablet. You would continue taking this medicine for a period of time, and the study will focus on what happens after 24 months (two years).

You would have regular visits to the clinic for assessments. These visits will involve blood tests to check your disease levels (molecular status), your general health, and to look for any side effects. Researchers will also ask about your quality of life to understand how the treatment affects you daily.

The actual duration of the medicine and follow-up visits would be explained in full by the study team, but the main goal is to assess your condition after two years of taking Iclusig.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit of this study is that Iclusig might help you achieve a very low level of CML, possibly leading to a chance to stop treatment in the future. However, there's no guarantee this will happen. Iclusig, like all medicines, can have side effects, and the research team will explain these to you in detail. You 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
Germany

Common questions

What is chronic myeloid leukaemia (CML)?

CML is a type of cancer that affects the blood and bone marrow, where the body makes new blood cells. It causes the body to make too many abnormal white blood cells.

What does 'MR4' mean?

MR4 stands for 'Molecular Response 4'. It means that very sensitive tests can no longer detect any signs of the CML disease in your body. It's a key step towards potentially stopping treatment.

What is 'treatment-free remission'?

This is when someone with CML has reached and maintained very low disease levels (like MR4) for a long time, allowing them to safely stop taking their CML medicine while remaining well.

What is Iclusig (ponatinib)?

Iclusig is a medicine approved to treat CML. It works by targeting the specific abnormal protein that causes CML.

Will I have to pay for the private medicine?

No, if you're accepted into the study, the study will cover the costs of the medication as part of the research.