Eculizumab in adults with atypical Haemolytic-Uraemic Syndrome

• Research type

  Research Study

• Full title

  AN OPEN-LABEL, MULTI-CENTER CLINICAL TRIAL OF ECULIZUMAB IN ADULT PATIENTS WITH ATYPICAL HAEMOLYTIC-URAEMIC SYNDROME

• IRAS ID

  60772

• Contact name

  Timothy H J Goodship

• Sponsor organisation

  ALEXION PHARMACEUTICALS, INC.

• Eudract number

  2010-020326-18

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  Atypical Haemolytic-Uraemic Syndrome (aHUS) is a serious and life-threatening disorder characterised by haemolytic anaemia (the abnormal breakdown of red blood cells), acute kidney failure and a low platelet (blood cells responsible for clotting) count (thrombocytopenia). These clinical abnormalities (thrombotic microangiopathy - TMA) are due to blood clots in the small blood vessels within the kidney microcirculation.The majority of patients with aHUS develop the disease before the age of 10. It may also first present itself in patients aged 20-40 years, but it can present at any age. The outlook for aHUS patients is poor ?? current medical therapy includes combating blood clotting (anticoagulants), suppressing the immune system, or balancing the lack of platelets clotting the blood (plasma therapy - PT). 50% of patients with aHUS require long-term dialysis and the disease almost invariably recurs after kidney transplant. Eculizumab has been approved for use in patients with another blood disease, Paroxysmal nocturnal hemoglobinuria (PNH), characterised by blood abnormalities similar to those seen in aHUS. Eculizumab is currently being tested in adults and adolescents with aHUS, with encouraging initial results.This study aims to confirm the effects of eculizumab in adult patients with aHUS to control the blood clots within the kidney microcirculation, and the safety of eculizumab.Participants of this study will receive 900mg eculizumab every week during the first 4 weeks of the induction phase followed by one dose of 1200mg for 1 week, and 1200mg of eculizumab during the maintenance phase every 2 weeks for a total treatment period of 2 years maximum. This is a multi-centre study, taking place in many hospitals and countries across Europe and other countries. It is anticipated that up to 30 adults worldwide will be enrolled in this study.

• REC name

  North East - Newcastle & North Tyneside 1 Research Ethics Committee

• REC reference

  10/H0906/67

• Date of REC Opinion

  19 Oct 2010

• REC opinion

  Favourable Opinion