Open-Label Dose Escalation and Safety Study in Haemophilia Patients

• Research type

  Research Study

• Full title

  An Open-label, Multi-centre, Dose-Escalation Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects with Haemophilia B.

• IRAS ID

  53146

• Contact name

  Steve Austin

• Sponsor organisation

  CSL Behring GmbH

• Eudract number

  2010-018477-38

• ISRCTN Number

  Not Known

• Research summary

  CSL Behring (CSLB) is studying a new recombinant coagulation Factor IX Albumin Fusion Protein (rIX-FP) in patients suffering from Haemophilia B. The purpose of this first in human study is to determine how long rIX FP remains in the blood and record side effects, if any, it may have. Haemophilia B is an inherited rare bleeding disorder in males caused by deficiency of Factor IX (FIX). Without sufficient FIX, the blood cannot clot properly to control bleeding and leads to an increased tendency for bleeding. Current commercially available FIX products need to be infused twice weekly to provide a temporary correction of FIX deficiency, thereby reducing disabling joint and tissue damage due to repeated bleeding. Based on information obtained from preclinical studies in animal, CSLB expects to demonstrate that rIX-FP has the benefit of being able to be given less often than standard FIX treatment. Approximately 20 patients worldwide will participate in the study - a dose escalation (25, 50 and 75 IU/kg) safety segment, and Pharmacokinetic evaluation segment of rIX-FP in comparison to a previous FIX product. Participants may participate in one or two segments (25/50IUkg or 50/75IUkg) over a 2-4 month period.

• REC name

  London - Riverside Research Ethics Committee

• REC reference

  10/H0706/76

• Date of REC Opinion

  28 Jan 2011

• REC opinion

  Further Information Favourable Opinion