Deferasirox in Non-Transfusion Dependant Thalassemia patients (THETIS)

• Research type

  Research Study

• Full title

  An open label, multi-center, efficacy and safety study of deferasirox in iron overloaded patients with non-transfusion dependent thalassemia (THETIS)

• IRAS ID

  108951

• Contact name

  John Porter

• Eudract number

  2012-000650-64

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  Thalassemia refers to a diverse group of genetic disorders that affect red blood cell production, causing anaemia. Unlike types of thalassemia in which patients require regular blood transfusions, NTDT patients can live without frequent transfusions. However, Iron Overload occurs in NTDT patients who have not been transfused because the body tells the gut to absorb more iron than usual from the food eaten. Transfusion, which brings healthy blood into the body, can also bring in extra iron. Over time this iron builds up in the liver because the body has no natural way to get rid of it. This buildup of excess iron in the liver over time can lead to liver damage. The THALASSA Trial which recently concluded shows deferasirox, an iron chelator, is effective in reducing liver iron levels in patients with Non-Transfusion-Dependent Thalassemia (NTDT). Deferasirox is expected to be approved for this indication in November 2012. This trial is designed to gain further information about deferasirox in the NTDT population and to see if will improve a patient??s endocrine function (i.e hormones) and quality of life. Approximately 117 patients will be enrolled from several countries and treatment duration will be 52 weeks. Patients will be asked to come to the hospital more frequently than normal to monitor their well-being. This trial is sponsored by the pharmaceutical company named Novartis.

• REC name

  South Central - Berkshire Research Ethics Committee

• REC reference

  12/SC/0436

• Date of REC Opinion

  5 Nov 2012

• REC opinion

  Further Information Favourable Opinion