NCT00941616

Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease

An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease.

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: CSL Behring · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD). Pharmacokinetic Component: PK parameters will be determined from a subgroup of subjects. Subjects who complete the PK component will subsequently continue in the efficacy component of the study, either continuing on a previously established prophylaxis regimen or continuing to receive on-demand treatment with the occurrence of non-surgical bleeding (NSB) events. Efficacy Component: Three treatment arms are defined for the efficacy component of the study. (1) Subjects who are currently being treated on a set prophylaxis regimen with a VWF product at the time of study entry will be enrolled in the "Prophylaxis" arm. (2) Subjects not being treated on a set prophylaxis regimen at the time of study entry who require a VWF product for the treatment of NSB events will be enrolled in the "On-demand" arm and commence using Biostate in the treatment of NSB events. (3) Subjects enrolled in the "On-demand" arm have the possibility to enter the "Cross-over to Prophylaxis" arm to receive an additional 12 months of prophylactic treatment.

Conditions

• Von Willebrand Disease

Interventions

Type	Name	Description
BIOLOGICAL	Biostate®	80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
BIOLOGICAL	Biostate®	Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.

Timeline

• Start date: 2009-06-01
• Primary completion: 2012-02-01
• Completion: 2012-02-01
• First posted: 2009-07-17
• Last updated: 2017-10-03

Locations

5 sites across 4 countries: Bulgaria, Poland, Russia, Ukraine