Eculizumab in children with atypical Haemolytic-Uraemic Syndrome

• Research type

  Research Study

• Full title

  An open-label, multi-centre clinical trial of eculizumab in paediatric patients with Atypical Haemolytic-Uraemic Syndrome (aHUS)

• IRAS ID

  64213

• Contact name

  Lesley Rees

• Sponsor organisation

  ALEXION PHARMACEUTICALS, INC.

• Eudract number

  2010-020310-28

• 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 (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 haemoglobinuria (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 paediatric patients with aHUS to control the blood clots within the kidney microcirculation, and the safety of eculizumab. Participants will receive different doses of the study drug depending on their body weight. The drug will be given weekly but the length of time during each stage will again depend on body weight. This is an open label (participants will know which dose they will be given), non-randomised, single-arm multicentre clinical trial of eculizumab in paediatric patients (1 month up to 18 years) with aHUS. The study will take place in many hospitals and countries across Europe and other countries. It is anticipated that up to 15 infants, children and adolescents worldwide will be enrolled in this study.

• REC name

  South Central - Berkshire Research Ethics Committee

• REC reference

  10/H0505/106

• Date of REC Opinion

  1 Feb 2011

• REC opinion

  Further Information Favourable Opinion