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PHAZAR trial

In this trial, researchers will investigate whether a new combination of drugs can be used to treat people with types of MPN.

The context

Myeloproliferative neoplasms (MPN) are disorders that cause the bone marrow to produce too many blood cells. It includes disorders such as: polycythaemia vera (PV), a disorder in which too many red blood cells are made, causing the blood to become thicker than normal, essential thrombocythaemia (ET), a disorder where there are too many platelets in the blood, which can cause the blood to clot, and myelofibrosis (MF), when the bone marrow is overactive, which causes scar tissue to develop (known as fibrosis).

The project

In this project, researchers want to see if two drugs called azacitidine and ruxolitinib can be given to people with MPN as a treatment. Both drugs are already used on their own to treat this group of people, but the trial wants to test how well they work when they are given together. Azacitidine is a type of chemotherapy that doctors use to treat people with AML and MDS. Its exact mechanism of action is unknown, and it has more than one effect on cells. In AML and MDS, important genes that regulate cell growth and division can be ‘switched-off’ when chemical tags stick to DNA inside cells and this can cause cells to grow out of control. Azacitidine is a drug that tries to reverse this by removing the chemical tags, switching on the gene again MPNs develop when processes going wrong inside cells. In particular, researchers think a process called ‘JAK signalling’ goes wrong in people with MPN and ruxolitinib might be able to reverse this to stop cancer cells from growing and dividing.

The aims

  • Find a safe dose of azactidine and ruxolitinib to have together
  • See how well treatment works
  • Find out more about the side effects