ALXN1210 vs eculizumab study in paroxysmal nocturnal haemoglobinuria

• Research type

  Research Study

• Full title

  A Phase 3, randomized, open-label, active-controlled study of ALXN1210 versus eculizumab in complement inhibitor-naïve adult patients with paroxysmal nocturnal hemoglobinuria (PNH)

• IRAS ID

  213108

• Contact name

  Anita Hill

• Contact email

  anitahill@nhs.net

• Sponsor organisation

  Alexion Pharmaceuticals

• Eudract number

  2016-002025-11

• Duration of Study in the UK

  2 years, 11 months, 13 days

• Research summary

  The purpose of this study is to determine whether study drug ALXN1210 is as good as eculizumab in its ability to effectively treat adult patients with Paroxysmal Nocturnal Haemoglobinuria (PNH) at less frequent dosing intervals. PNH is a condition caused by an abnormality that causes destruction of red blood cells. A drug similar to ALXN1210, called eculizumab, is approved for the treatment of PNH in 46 countries under the brand name Soliris®.

  ALXN1210 is being developed for the treatment of disorders involving the complement system. The complement system is a part of the complex immune system (system that fights against infections). ALXN1210 works by suppressing the activity of a specific portion of the complement system; it is therefore classified as an immunosuppressant drug. PNH is an example of a complement disease that could be eventually treated with ALXN1210. Participants who have not had any previous treatment with a complement inhibitor are eligible.

  Patients will be placed into 1 of 6 groups based on their previous transfusion history and LDH levels. Patients will then be randomised to receive either eculizumab or ALXN1210. The dose of ALXN1210 a patient will receive will be decided based on the patients weight. The study drug will be administered intravenously into a vein and blood samples will be collected throughout the study for various analysis either prior to, or after, study drug administration.

  There will be 3 periods in this study: Screening Period, Randomised Treatment Period and Extension Period. The duration of participation in the study is approximately 2.6 years.

  It is expected that approximately 300 centres will be opened around the world in order to enrol 214 participants for evaluation.

• REC name

  North West - Haydock Research Ethics Committee

• REC reference

  16/NW/0737

• Date of REC Opinion

  2 Dec 2016

• REC opinion

  Further Information Favourable Opinion