Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Randomized, Open-label, Phase 2 Trial of Ponatinib in Patients with Resistant Chronic Phase Chronic Myeloid Leukemia to Characterize the Efficacy and Safety of a Range of Doses

This research study is for adults with chronic myeloid leukaemia (CML) in the chronic phase, specifically when their previous treatments haven't worked well. Scientists are testing different doses of a medicine called Iclusig (also known as ponatinib) to see how effective and safe it is. They want to find out the best dose to reduce the signs of CML in the blood and bone marrow, and how long these effects last. The study will also carefully check for any side effects. By learning more about Iclusig, doctors hope to offer better treatment options for people living with CML that is resistant to other therapies.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Takeda Development Center Americas Inc.

Enrolment target

Start

03 Oct 2024

Chronic Phase Chronic Myeloid Leukemia

What is this study about?

This study is looking into a medication called Iclusig, also known by its active ingredient ponatinib, for people in the UK who have a specific type of blood cancer called chronic myeloid leukaemia (CML). CML is a long-lasting condition where too many white blood cells are made in the bone marrow. This particular study is for people whose CML hasn't responded well to other treatments, meaning their cancer cells are still present or growing despite previous efforts.

The main goal is to understand how different doses of Iclusig work. Researchers will be giving participants various doses – 15mg, 30mg, or 45mg tablets – to see which dose is most effective at reducing the number of CML cells, measured by a special test called BCR-ABL1IS. They also want to make sure the chosen doses are safe and don't cause too many serious side effects. Over a period of time, they will carefully monitor how participants respond to the treatment and keep track of any changes in their health.

By taking part, you would be helping doctors and scientists learn more about Iclusig. This information is really important because it could lead to improved care and new treatment options for people with CML that is difficult to treat. The study aims to gather detailed information about how well Iclusig works, how long its effects last, and what its side effects might be, ultimately contributing to better management of this condition.

Key takeaways

This study is for adults with CML that hasn't responded to previous treatments.
It's testing different doses of the medicine Iclusig (ponatinib).
The main goals are to find the best dose and check how well it works and if it's safe.
You would take Iclusig tablets and have regular check-ups.
Participation could offer a new treatment option for resistant CML.

Who may be eligible?

This study is for adults, aged 18 years and older, who have chronic myeloid leukaemia (CML) in its chronic phase. This means your CML has not progressed to a more advanced stage, but it hasn't responded as well as doctors hoped to previous treatments. Both men and women can take part.

To be considered for this study, your doctor will need to confirm that you meet several specific medical criteria related to your CML and overall health. These checks are important to make sure the study medication is appropriate and safe for you, and that the study results are clear.

Some things might mean you can't join, such as having certain other health conditions, or if your CML has progressed to a more aggressive form. Your medical team will go through all these details with you to see if this study is a good fit.

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) in the chronic phase?
Has your CML not responded well to previous treatments?
Are you able to attend regular hospital or clinic appointments?
Are you generally in good health, apart from your CML, as determined by a doctor?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would primarily be taking a medicine called Iclusig (ponatinib) in tablet form, typically once a day. The dose you receive would be determined by the study doctors, and could be 15mg, 30mg, or 45mg. You would have regular visits to your hospital or clinic. During these visits, doctors and nurses would perform various checks. This includes taking blood samples to monitor your CML and check for side effects, as well as general health checks like measuring your blood pressure and weight.

These check-ups are very important to make sure the treatment is working as expected and to quickly identify any side effects. The study will follow your progress closely over many months, potentially up to two years or more, to understand the long-term effects of the medication. The exact number of visits and tests will be explained in detail, but you can expect frequent monitoring, especially in the beginning.

Potential risks and benefits

Taking part in this study might offer you access to a new treatment option, Iclusig, for your CML that hasn't responded to other medicines. This could potentially help reduce the signs of your cancer. However, like all medicines, Iclusig can have side effects, which might include anything from mild discomfort to more serious reactions. The study team will monitor you very closely for any side effects. You are free to stop participating in the study at any time, for any reason, without it affecting your usual medical care. This choice is entirely yours throughout the study.

Locations (2)

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

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Unverified
Poland
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Unverified
France

Common questions

What is Chronic Myeloid Leukaemia (CML)?

CML is a type of cancer that affects the blood and bone marrow. It causes the body to make too many white blood cells, which don't work properly.

What does 'resistant' CML mean?

Resistant CML means that your cancer hasn't responded well to the treatments you've had in the past, or it has stopped responding to them.

What is Iclusig (ponatinib)?

Iclusig is a medicine used to treat CML. It works by targeting specific proteins that help the cancer cells grow, aiming to stop them from multiplying.

Why are there different doses of Iclusig in the study?

The study aims to find the best dose of Iclusig that is effective in treating CML while also being as safe as possible for patients. Testing different doses helps achieve this.

Will I know which dose I am receiving?

Yes, this is an 'open-label' study, which means both you and your study doctor will know which dose of Iclusig you are receiving.