Chief investigator - Dr Alexander Sternberg, University of Oxford
A phase Ib study of eltrombopag and azacitidine in patients with high risk myelodysplastic syndromes and related disorders
Award start date: 01 Oct 2012
Recruitment start date: 15 Oct 2014
Award duration: 5 years (59 months)

MDS is a serious disorder of blood cell production that causes fatigue, infection and bleeding. Certain types of MDS ('high risk' MDS) are prone to change their character and become cancerous - resulting in leukaemia.

Azacitidine is a type of chemotherapy drug that doctors already 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 are turned off by methyl groups becoming attached to the cells’ DNA, allowing cells to grow out of control. Azacitidine can act as a demethylating agent, removing the methyl groups and restoring normal gene function.

A recent trial has shown that a chemotherapy called azacitidine improves survival in people with high risk MDS. But many people in this trial had to have their azacitidine reduced or stopped because of low platelet counts and bleeding.

Researchers want to see a drug called eltrombopag helps to keep the platelet count up during azacitidine treatment. Although eltrombopag has been used for other disorders, doctors do not know what the correct dose eltrombopag should be for people with high risk MDS patients being treated with azacitidine.

The aims of the trial are to:

  • Find out the highest, safest dose of eltrombopag to give with azacitidine
  • Find out what the side effects are of having these two drugs together
  • See if the treatment helps people with MDS that has come back after a stem cell transplant

You may be eligible to join this trial if:

  • You have a low number of platelets
  • You are at least 16 years old