Deferasirox in non transfusion Thalassemia patients with iron overload

• Research type

  Research Study

• Full title

  A randomized, double-blind, placebo-controlled, phase II study to evaluate efficacy and safety of deferasirox in nontransfusion-dependent thalassemia patients with iron overload.

• IRAS ID

  6257

• Sponsor organisation

  Novartis - Bertrand Rabaut

• Eudract number

  2007-007000-15

• ISRCTN Number

  n/a

• Research summary

  Thalassaemia is an inherited blood disorder causing shortened red cell survival and anaemia . This results in a lack of oxygen supply to organs and triggers excessive iron uptake through the gut. This results in iron overload in the body, causing damage to internal organs. Small molecules known as Ó?iron chelators? bind to excess iron and remove it from the body.Severe Thalassaemia patients require regular blood transfusions, however as haemoglobin already contains iron, transfusions further increase iron overload. Patients as severe as these are given iron chelators in order to eliminate toxic levels of iron in the body.This study focuses on a group of milder patients that don??t depend on regular blood transfusions. Their cases are not as severe, however still suffer iron overload, due to the ineffective production of red blood cells that stimulates increased iron absorption. Iron overload in these patients leads (later on in life) to the same complications as those more severe cases. There is no current approved treatment for these patients.This study aims to evaluate the safety and effects of Deferasirox in Non Transfusion Dependant Thalassaemia patients. Deferasirox is an iron chelator (produced in a tablet) given to severe Thalassaemia patients and will be tested on this milder patient group over a 12 month period. Deferasirox or Placebo (dummy drug containing no active ingredient) will be given to patients once daily with the aim of reducing iron overload and preventing further iron build up in the body. Patients will attend the clinic 19 times over 12 months and will undergo specific assessments measuring general health and iron levels as a result of taking Deferasirox compared to placebo.

• REC name

  London - Harrow Research Ethics Committee

• REC reference

  08/H0714/89

• Date of REC Opinion

  19 Jan 2009

• REC opinion

  Further Information Favourable Opinion