Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment of Bleeding Episodes in Von Willebrand Disease (VWD)

Summary

The purpose of this Phase 3 study is to assess the pharmacokinetics of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary von Willebrand disease (VWD).

Timeline

Start

2011-11-01

Primary completion

2014-02-01

Completion

2014-02-01

Outcome

Met primary endpoint

paper Treatment success, defined as the number of subjects with a mean efficacy rating of <2.5, was 100%. PMID 26239086 ↗

registry NUMBER Full Analysis Set: 100.0 (84.7 to 100.0) Percent of participants NCT01410227 ↗

Publications

• Results Gill JC, Castaman G, Windyga J, Kouides P, Ragni M, Leebeek FW, Obermann-Slupetzky O, Chapman M, Fritsch S, Pavlova BG, Presch I, Ewenstein B. Hemostatic efficacy, safety, and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease. Blood. 2015 Oct 22;126(17):2038-46. doi: 10.1182/blood-2015-02-629873. Epub 2015 Aug 3.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Recombinant Factor VIII	Protein / enzyme biologic	—	Intravenous
Subject	Recombinant von Willebrand Factor	Protein / enzyme biologic	50 iu/kg	Intravenous
Subject	Recombinant von Willebrand Factor	Protein / enzyme biologic	80 iu/kg	Intravenous

Indications