Phase II, combo study with Exjade/Desferal- cardiac iron overload

• Research type

  Research Study

• Full title

  Phase II, open-label, single-arm, multicenter study to evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed by deferasirox monotherapy in patients with severe cardiac iron overload due to chronic blood transfusion (HYPERION)

• IRAS ID

  62705

• Contact name

  John Porter

• Eudract number

  2010-021062-29

• ISRCTN Number

  n/a

• Research summary

  This study, HYPERION, will evaluate the effect of a combination therapy of two medicines in patients who have severe iron overload in the heart due to chronic blood transfusions.The first medicine is called deferisirox (also code named ICL670 and known as Exjade) and the second medicine is called deferoxamine (also known as DFO or Desferal).Deferisirox (ICL670) and Deferoxamine (DFO) have been approved by UK health authority and by other health agencies around the world for the treatment of excess iron due to blood transfusions. Both drugs are also commercially available.Both medicines (when givenoseparately) have been shown to adequately remove iron from the body, as indicated by results from studies that looked at their effects on iron content in the liver and in the heart. In this study, the once a day oral suspension of ICL670 will be combined with a 5 times a week DFO infusion during at least 6 months. The level of iron in the heart will be measured by specific assessments every 6 months. Once this level has decreased, patients will be treated only by oral ICL670 until the end of the study. If it increases again, the doctor would be able to give again the combination therapy before the end of the study.It is planned that 45 patients will join this study at approximately 20 study sites in about 10 countries within Europe, Latin America, Asia and Middle East. Participants will be asked to come to the hospital at least every month for 24 months to undergo regular clinical examination, MRI scans (provides very detailed images of the internal organs), ECHO (produces image of the heart), ECG (recoris how the heart beats), eye test assessments and laboratory assessments.This clinical study is sponsored by a pharmaceutical company named Novartis.

• REC name

  London - Harrow Research Ethics Committee

• REC reference

  10/H0715/64

• Date of REC Opinion

  21 Dec 2010

• REC opinion

  Further Information Favourable Opinion