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This trial will look at a new treatment combination for people with a more advanced phase of CML who have limited treatment options left.

The context

Chronic myeloid leukaemia (CML) is a type of blood cancer that affects certain types of your white blood cells. CML cells produce a protein called BCR-ABL that isn't found in healthy cells. This protein can cause these cancerous cells to grow out of control. People with CML are often treated with drugs which block this protein including drugs such as imatinib or nilotinib to prevent these cancerous cells growing out of control. These drugs have improved the outcome for people with CML dramatically. However, they don’t work for everyone with the disease and some people will require treatment including chemotherapy and stem cell transplant.

The project

This trial is looking at a new treatment combination which is made up of a chemotherapy regime called FLAG-IDA and another drug which targets the BCR-ABL mutation called ponatinib. This will be tested in people with more CML, who have few other treatment options available to them. Ponatinib works in a similar way to imatinib and nilotinib, but it was also designed to work in cancers which no longer respond to these drugs. If found to be safe, researchers hope that this combination of drugs can be used in future trials to see whether we can improve the outlook for people with more advanced CML.

The aims

  • Find the best dose of ponatinib for people having intensive treatment for CML that is at a more advanced stage, called the ‘blast phase’
  • See how well ponatinib works for this group of people
  • Learn more about the side effects of this drug