Protein-free FVIII (BAY 81-8973)in subjects with severe haemophilia A

• Research type

  Research Study

• Full title

  A two part, randomized, cross-over, open-label trial to evaluate the pharmacokinetics, efficacy, and safety profile of plasma protein-free recombinant FVIII formulated with sucrose (BAY 81-8973) in previously treated subjects with severe hemophilia A under prophylaxis therapy.

• IRAS ID

  60508

• Contact name

  Kingsley Hampton

• Sponsor organisation

  Bayer Plc

• Eudract number

  200901214943

• Clinicaltrials.gov Identifier

  NCT01029340

• Research summary

  Congenital haemophilia A is a genetic disorder affecting mostly male individuals, in which one of the proteins needed to form blood clots is missing or not present in sufficient levels. In a person with haemophilia A, the clotting process is much slower and the person experiences bleeding episodes that can result in serious problems and potential disability.Recombinant coagulation Factor VIII (rFVIII) is the leading treatment for patients with haemophilia A. Bayer HealthCare has produced 2 rFVIII products, KOGENATE© and its successor KOGENATE© Bayer. BAY81-8973 is a new full length recombinant human FVIII which will be the successor of Kogenate Bayer. BAY81-8973 is produced by a new and improved manufacturing process which is run without any added constituents from human plasma and animal materials and a new nanofiltiltration step has been added.. The result of the technical improvement in the production of BAY81-8973 is a full length rFVIII that consistently reflects the structure of the native human FVIII protein.The study is composed in two parts: Part A (Phase I only) and Part B (Phase II/III). Part A will assess how the body affects BAY 81-8973 (pharmacokinetics) compared to Kogenate Bayer. Part B will assess the safety, tolerability, and efficacy of prophylaxis treatment (2 to 3 times a week) with BAY 81-8973 in 60 subjects over a one year period (split into two six month treatment periods). The UK will only participate in Part B.Sixty subjects will be randomized to one of 2 treatment arms and receive open-label prophylactic administration of BAY 81 8973. There are 2 different methods measuring the amount of study drug (assays), the study will compare the chromogenic substrate assay per European Pharmacopeia (CS/EP) with the classical assay (Chromogenic Substrate Adjusted, CS/ADJ). During one six month period patients will receive the study drug where the dose has been measured using the? (CS/EP) and during the other six months period the dose will be measured based on the Chromogenic Substrate Adjusted assay CS/ADJ).

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  10/H0405/92

• Date of REC Opinion

  26 Nov 2010

• REC opinion

  Further Information Favourable Opinion