Eculizumab in adults with PT-sensitive aHUS

• Research type

  Research Study

• Full title

  An open-label, multi-centre controlled clinical trial of Eculizumab in adult patients with plasma therapy-sensitive atypical Haemolytic-Uremic Syndrome (aHUS)

• IRAS ID

  18177

• Contact name

  Tim Goodship

• Sponsor organisation

  Alexion Pharmaceuticals, Inc

• Eudract number

  2008-006954-17

• ISRCTN Number

  N/A

• Research summary

  Atypical Haemolytic-Uremic Syndrome (aHUS) is a serious and life-threatening disorder characterised by haemolytic anaemia (the abnormal breakdown of red blood cells), acute renal failure and a low platelet count (thrombocytopenia). These clinical abnormalities are due to blood clots (thrombotic microangiopathy [TMA]) in the small blood vessels within the kidney microcirculation.The majority of patients with aHUS develop the disease before age 10, though it may also first present itself in patients aged 20-40 years.Prognosis for aHUS patients is poor - and current therapy includes combating blood clotting (anticoagulants), suppressing the immune system, supportive care or plasma therapy (PT). 50% of patients with aHUS require long-term dialysis, and 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. In this disorder, eculizumab was shown to reduce the destruction of red blood cells (haemolysis). Five patients with aHUS have also been treated with eculizumab, with encouraging initial results. In this study, the effects of eculizumab on the blood clots within the kidney microcirculation will be confirmed by the absence of; 1. a decrease in platelet count; 2. PT whilst receiving eculizumab; 3. new dialysis for at least 12 weeks.All patients in this study will be assigned to a single dosing regime. After an initial 8 week observation period, patients will receive 900mg of eculizumab, given once a week intravenously, for 4 weeks. This will then increase to 1200mg of eculizumab, which will continue to be given every 14 days for another 22 weeks. After the treatment period, patients will continue to attend follow-up appointments at various intervals for another 8 weeks.This is a multi-centred study, taking place in many hospitals and countries across Europe and North America. It is anticipated that 15 adults worldwide will be enrolled in this study, 4 of these being from the UK.

• REC name

  North East - Newcastle & North Tyneside 1 Research Ethics Committee

• REC reference

  09/H0906/43

• Date of REC Opinion

  28 May 2009

• REC opinion

  Further Information Favourable Opinion