Single arm study of Safety and Efficacy of Coversin in adult aHUS

• Research type

  Research Study

• Full title

  A Phase 2 single arm study of Safety and Efficacy of Coversin in adult aHUS subjects

• IRAS ID

  231506

• Contact name

  Marie Scully

• Contact email

  m.scully@ucl.ac.uk

• Sponsor organisation

  Akari Therapeutics Plc

• Eudract number

  2017-000064-15

• Duration of Study in the UK

  2 years, 0 months, days

• Research summary

  Atypical haemolytic uremic syndrome (aHUS) are extremely rare, life-threatening disease of the blood, characterised by the destruction of red blood cells by the immune system.
  Breakdown of the red blood cells causes blood clots and anaemic symptoms such as fatigue. aHUS is caused by one or several mutations in genes that result in uncontrolled and excessive activation of the ‘complement’ system (a part of the body’s immune system). High risk of blood clots results in renal failure in most patients.
  Most therapies for aHUS aim to ease symptoms. Iron supplements and folic acid are usually given to patients to offset the high turnover of red blood cells. Blood transfusions may be required, or warfarin to reduce the risk of blood clots however both have risks and are not always effective. The only current therapy for aHUS is Eculizumab which inhibits the complement system, however this medication must be given by a healthcare professional every 2 weeks. There is an unmet need for the use of a more convenient treatment with fewer risks.

  Akari Therapeutics PLC have developed a new medication, Coversin, that has been designed to inhibit the complement
  system.
  This study is being done to demonstrate the safety and efficacy of Coversin in patients who have relapsed or have been newly diagnosed aHUS.
  This is an open-label, multicentre study which will take place across Europe, involving approximately 10 patients.
  Participants who react well to Coversin will continue to receive Coversin treatment if they choose to participate and meet eligibility criteria for the extension study for another 3 years or until product is licensed and reimbursed.

• REC name

  East of Scotland Research Ethics Service REC 2

• REC reference

  17/ES/0107

• Date of REC Opinion

  14 Sep 2017

• REC opinion

  Further Information Favourable Opinion