A Prospective Phase II Clinical Trial Evaluating the Efficacy and the Safety of Tyrosine Kinase Inhibitors Withdrawal After a Previous Two-step Dose Reduction in Patients With Chronic Myeloid Leukemia in Deep Molecular Remission
Chronic myeloid leukaemia (CML) is a type of blood cancer usually treated with daily targeted tablets called tyrosine kinase inhibitors (such as Glivec, Sprycel, or Tasigna). Many people respond so well to these medicines that tests find barely any leukaemia cells left in their blood. This state is known as deep molecular remission. Traditionally, patients continue taking these tablets for the rest of their lives. However, daily treatment can cause long-term side effects such as joint pain and fatigue. This study tests whether it is safe for patients in deep remission to lower their tablet dose in two stages before stopping treatment completely. Doctors will track participants closely with regular blood tests over three years. If leukaemia markers rise, treatment is restarted straight away. The main goal is to see if patients can safely live medicine-free while maintaining a good quality of life.
At a glance
What is this study about?
Chronic myeloid leukaemia (CML) is a slow-growing cancer of the white blood cells. For many years, people with CML have relied on daily tablets called tyrosine kinase inhibitors (or TKIs, including imatinib, nilotinib, and dasatinib). These medicines work remarkably well. They often push leukaemia levels down to tiny, almost undetectable amounts, allowing people to live long and active lives.
Even though these medicines are life-saving, taking them every day can lead to lasting side effects such as tiredness, muscle aches, and digestive problems. In the past, doctors believed patients needed to stay on these tablets forever. Today, specialists want to find out whether patients with deep, long-lasting remission can safely reduce their dose and eventually stop taking the medicine altogether.
This clinical trial tests a careful, step-by-step approach. Rather than stopping the tablets all at once, participants will reduce their dose in two planned steps before pausing treatment completely. Highly sensitive blood tests will be carried out at regular intervals to check for any sign of leukaemia cells returning.
If the cancer markers start to climb, participants restart their standard medication immediately to bring the leukaemia back under control. By testing this gradual reduction, researchers hope to give more people the chance to safely stop treatment, avoid side effects, and enjoy life without daily cancer pills.
Key takeaways
- Tests whether adults with CML can safely lower their daily tablet dose and then stop taking it completely.
- Open to adults aged 18 and older who are in deep, stable molecular remission.
- Uses a gradual two-step dose reduction before stopping medication altogether.
- Patients are protected by regular, sensitive blood tests over a three-year period.
- Standard medication is restarted straight away if leukaemia markers show any sign of rising.
- Aims to reduce long-term side effects and improve day-to-day quality of life.
Who may be eligible?
This study is open to adults aged 18 and over who have been diagnosed with chronic myeloid leukaemia (CML). To be considered, participants must currently be taking a standard targeted tablet (such as Glivec, Tasigna, or Sprycel) and have achieved a 'deep molecular response'. This means specialised blood tests show that leukaemia cells have dropped to extremely low, barely detectable levels.
The trial is not suitable for people whose leukaemia is still active or unstable, or for those whose blood test markers have fluctuated recently. Your hospital doctor will review your medical history and recent blood test records to see if your remission is deep and stable enough to make stopping medication safe 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.
- I am aged 18 or older.
- I have been diagnosed with chronic myeloid leukaemia (CML).
- I currently take a daily targeted tablet such as Glivec, Tasigna, or Sprycel.
- My doctor has confirmed that my leukaemia is in deep molecular remission.
- I am able and willing to attend regular hospital appointments for blood tests.
What does participation involve?
Taking part in this study lasts for up to 36 months (three years). At first, your doctor will lower your daily tablet dose in two planned steps while checking your blood results closely. If your leukaemia remains undetectable, you will then stop taking the tablets entirely. Throughout the study, you will visit the hospital regularly for blood tests that track cancer markers, blood sugar, and cholesterol levels. If your blood tests ever show that leukaemia levels are rising, you will be restarted on your regular medication right away to regain deep remission.
Potential risks and benefits
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Common questions
What happens if my leukaemia levels begin to rise?
If your regular blood tests show that leukaemia markers are increasing, your doctor will restart your standard tablets immediately. Past research shows that the vast majority of patients regain deep remission quickly once treatment resumes.
What is targeted therapy withdrawal syndrome?
When people stop taking these daily tablets, some develop temporary joint, bone, or muscle pain. These symptoms usually settle within a few weeks or months, and your doctor can recommend simple pain relief if needed.
Will taking part in this trial cost me anything?
No. All study-related hospital visits, blood tests, and consultations are provided as part of the clinical trial without cost to you.
How often will I need to have blood tests?
You will need frequent blood tests—often every few weeks to months, particularly in the first year after lowering or stopping your dose—to ensure any change in your condition is caught very early.
Can I leave the study if I feel anxious about not taking tablets?
Yes. You have the right to withdraw from the trial at any point, for any reason, and you can discuss restarting your regular medication with your specialist.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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