Deferasirox for early iron loading in transfusion-dependant MDS

• Research type

  Research Study

• Full title

  A phase 2 study of the efficacy and safety of Deferasirox administered at early iron loading in patients with transfusion-dependent Myelodysplastic Syndromes.

• IRAS ID

  103687

• Contact name

  Dominic Culligan

• Sponsor organisation

  University of Birmingham

• Eudract number

  2011-004559-38

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  Myelodysplastic Syndromes (MDS) cause a failure of the bone marrow, which does not produce enough blood cells (red cells, white cells and platelets). This is because the bone marrow contains too many abnormal cells (dysplastic cells) which function poorly. Many patients with MDS, do not produce enough red blood cells, which leads to anaemia. This means that they receive regular blood transfusions to treat the anaemia and alleviate symptoms. However, blood is rich in iron and repeated transfusions may cause a build-up of excess iron. Although iron is an essential part of the blood, an excess of iron may affect the way the organs in the body function. This includes the liver and heart. This situation is called iron-overload. The aim of this study is test how effective, safe and tolerable a drug called Deferasirox (also called Exjade©) is, when used to treat rising iron levels, in patients with MDS. The study treatment will aim to control the iron levels in the blood, which steadily increase after receiving regular blood transfusions. It is not intended to treat MDS. Normally doctors will wait until the level of iron in the blood significantly increases before considering starting treatment for iron-overload, but in this study we would like to give Deferasirox treatment early rather than waiting for the iron levels to rise until a high level is reached and organ damage begins. In summary, we are looking at the feasibility of starting treatment early, before iron overload begins.

• REC name

  North East - Newcastle & North Tyneside 1 Research Ethics Committee

• REC reference

  12/NE/0220

• Date of REC Opinion

  26 Jul 2012

• REC opinion

  Further Information Favourable Opinion