AuthorisedTherapeutic exploratory (Phase II)Interventional

A phase II, multicenter, open-label, single arm study to evaluate the safety and efficacy of asciminib in pediatric participants newly diagnosed or previously treated with Philadelphia positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) with or without known T315I mutation

This study is testing a new medicine called asciminib for children and young people who have a type of blood cancer known as Philadelphia positive Chronic Myeloid Leukaemia (CML) in the chronic phase. This is a type of blood cancer that affects the bone marrow, where blood cells are made. Researchers want to find out how safe asciminib is and how well it works. They are particularly interested in whether it can help patients achieve a 'major molecular response' (MMR), which means a significant reduction in the amount of leukaemia cells in their body. This study includes patients newly diagnosed or those who haven't responded well to previous treatments. All participants will receive asciminib.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Novartis Pharma AG

Enrolment target

Start

18 May 2026

Philadelphia positive Chronic Myeloid leukemia in Chronic Phase with newly diagnosed and resistant/intolerant to previous TKIs, with or without the T315I mutation

What is this study about?

This study is about a new medicine called asciminib. It's being tested for children and young people who have a particular type of blood cancer called Philadelphia positive Chronic Myeloid Leukaemia (CML) in its chronic phase. CML is a type of cancer that starts in the bone marrow, the soft tissue inside your bones where blood cells are made. In Philadelphia positive CML, there's a specific change in the chromosomes within the cancer cells, often called the 'Philadelphia chromosome'.

The main goal of this study is to see how safe this new medicine, asciminib, is for these young patients and how well it works. The researchers want to know if it can help reduce the amount of leukaemia cells in the body, which doctors call a 'major molecular response' (MMR). They will be checking for this response after 48 weeks of treatment.

This study is open to patients who have recently been diagnosed with this type of CML, as well as those who have been treated before but whose leukaemia has not responded well to their previous medicines, or who had side effects they couldn't live with. By studying asciminib, doctors hope to find a new and effective treatment option for children and young people living with Philadelphia positive CML.

Key takeaways

This study is testing a new medicine for young people with a specific type of blood cancer (Philadelphia positive CML).
It aims to see how safe the medicine is and if it can significantly reduce cancer cells.
It includes both newly diagnosed patients and those who haven't responded to previous treatments.
All participants will receive the new medicine, asciminib.
Regular health checks and blood tests will be part of the study participation.

Who may be eligible?

This study is for children and young people diagnosed with a specific type of blood cancer called Philadelphia positive Chronic Myeloid Leukaemia (CML) in its 'chronic phase'. This includes patients who have just been diagnosed with this condition.

It also includes patients who have been treated for this leukaemia before but haven't responded well to their previous medicines. This means their leukaemia either didn't get better, or it came back, or they had side effects from other treatments that were too difficult to manage.

Both boys and girls of any age are being considered for this study. The medical team will check your specific situation to see if this study is the right fit 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.

Do I have Philadelphia positive Chronic Myeloid Leukaemia (CML)?
Is my CML in the 'chronic phase'?
Am I newly diagnosed, or have my previous CML treatments not worked well or caused too many side effects?
Has my doctor confirmed that this study might be suitable for me?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive the study medicine, asciminib. You will have regular visits to the clinic where doctors and nurses will check your health, take blood and other samples, and ask about any side effects you might be experiencing. They will carefully monitor your leukaemia to see how well the treatment is working.

These check-ups will happen at specific times throughout the study. They will include tests like blood tests to look at your blood cell counts and the levels of the leukaemia cells, and possibly bone marrow tests. For children and adolescents, there will also be checks on growth, such as height, weight, and bone age (using an X-ray), and how they are developing physically. The total duration of your participation will depend on how you respond to the treatment and the study plan, but the main results will be looked at after 48 weeks and then again at 96 weeks.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. The potential benefit of this study is that asciminib might be an effective treatment for your leukaemia, helping to reduce the amount of cancer cells in your body and improve your health. However, like all medicines, asciminib can have side effects, which the medical team will explain to you and monitor closely. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (6)

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

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Unverified
Spain
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Unverified
France
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Unverified
Germany
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Unverified
Poland
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Unverified
Italy
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Unverified
Netherlands

Common questions

What is Philadelphia positive CML?

It's a type of blood cancer where there's a specific change in the DNA of your blood cells, called the Philadelphia chromosome, causing your body to make too many white blood cells.

What does 'major molecular response' mean?

This means there's been a significant drop in the number of leukaemia cells in your body, as measured by very sensitive tests. It's a sign the treatment is working well.

Will I get a placebo (dummy medicine)?

No, in this study, all participants will receive the study medicine, asciminib.

What are 'side effects'?

Side effects are unwanted effects of a medicine. The study team will monitor you closely for any side effects and help manage them.

How long will I be in the study?

The main results are looked at after 48 weeks and 96 weeks, but your individual participation might vary based on your response and the study doctor's advice.