Clinical trials and chronic leukaemia

Clinical trials are carried out to try to find new and better treatments for chronic myeloid leukaemia (CML) and chronic lymphocytic leukaemia (CLL).

What are clinical trials?

Trials that are carried out on patients are known as clinical trials. These may be carried out to:

  • test new treatments, such as new chemotherapy drugs or targeted therapies
  • look at new combinations of existing treatments, or change the way they are given to make them more effective or reduce side effects
  • compare the effectiveness of drugs used to control symptoms
  • find out how leukaemia treatments work
  • find out which treatments are the most cost-effective.

Trials are the only reliable way to find out if a different type of treatment is better than what is already available.

Clinical trials are carried out to try to find new and better treatments for chronic myeloid leukaemia (CML) and chronic lymphocytic leukaemia (CLL).

Taking part in a clinical trial

You may be asked to take part in a treatment clinical trial. There can be many benefits to this. Trials help to improve knowledge about leukaemia and develop new treatments. You will be carefully monitored during and after the study.

Usually, several hospitals around the country take part in these trials. But some treatments that look promising at first are later found not to be as good as existing treatments, or have side effects that outweigh the benefits. This is something for you to think about.

If you decide not to take part in a trial, your decision will be respected and you do not have to give a reason. There will be no change in the way you are treated by the hospital staff, and you will be offered the standard treatment for your situation.

Blood and biopsy samples

Blood and biopsy samples may be taken to help make the right diagnosis. You may be asked for your permission to use some of your samples for research into leukaemia.

If you take part in a trial you may also give other samples, which may be frozen and stored for future use when new research techniques become available. Your name will be removed from the samples so you cannot be identified. The research may be carried out at the hospital where you are treated, or at another one.

This type of research takes a long time, and results may not be available for many years. The samples will be used to increase knowledge about the causes of leukaemia and its treatment, which will hopefully improve the outlook for future patients.

About our information

  • CML References

    Below is a sample of the sources used in our chronic myeloid leukaemia (CML). If you would like more information about the sources we use, please contact us at

    European Leukemia Net. Recommendations for the management of chronic myeloid leukemia. 2013.

    Hoffbrand V, and Moss P. Hoffbrand’s essential haematology. 7th edition. 2016.

    National Institute for Health and Care Excellence. Leukaemia (chronic myeloid) – dastatinib, nilotinib and standard dose imatinib for the first-line treatment of chronic myeloid leukaemia (part review of technology appraisal guidance 70). April 2012.

    National Institute for Health and Care Excellence. Technology appraisal guidance. 401/426/425.

    DeVita V, Lawrence T and Rosenberg S. 2016. Lymphomas and leukemias. From Cancer: principles and practice of oncology.

  • Reviewers

    This information has been written, revised and edited by Macmillan Cancer Support’s Cancer Information Development team. It has been reviewed by expert medical and health professionals and people living with cancer. It has been approved by Senior Medical Editors, Dr Anne Parker, Consultant Haematologist; and Dr Helen Marr, Consultant Haematologist.

    Our cancer information has been awarded the PIF TICK. Created by the Patient Information Forum, this quality mark shows we meet PIF’s 10 criteria for trustworthy health information.