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

Von Willebrand Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00941616
• Other study ID #: CSLCT-BIO-08-54
• Secondary ID: 14812008-004922-
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: July 15, 2009
• Last updated: October 2, 2017
• Start date: June 2009
• Est. completion date: February 2012

Study information

• Verified date: October 2017
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: February 2012
• Est. primary completion date: February 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Diagnosed with VWD

• Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not available

• Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B) within 10 years prior to their first dose of Biostate®

• Written informed consent given

Exclusion Criteria (for participation in the PK component):

• Actively bleeding immediately prior to initial PK period

• Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product

• Have Type 2B, 2N or 2M VWD

Exclusion Criteria (for all subjects):

• Requiring a VWF product for a planned surgical procedure at enrolment

• Have received aspirin or other non-steroidal anti-inflammatory drugs within 7 days prior to their first dose of study product

• Known history of, or are suspected to have, VWF or FVIII inhibitors

• Suffering an acute or chronic medical condition, other than VWD, which may affect the conduct of the study

• Known or suspected hypersensitivity or previous evidence of severe side effects to Biostate®, VWF/FVIII concentrates, or human albumin

• Impaired liver function at screening

• Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit

• Participation in a clinical study or use of an investigational compound in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period.

• Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening

Related Conditions & MeSH terms

• Von Willebrand Disease
• Von Willebrand Diseases

Intervention

Biological:
• Biostate®
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
• 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.

Locations

Country	Name	City	State
Bulgaria	Study Site	Sofia
Poland	Study Site	Warsaw
Poland	Study Site	Wroclaw
Russian Federation	Study Site	Barnaul
Ukraine	Study Site	Lviv

Sponsors (2)

Lead Sponsor	Collaborator
CSL Behring	Parexel

Countries where clinical trial is conducted

Bulgaria,  Poland,  Russian Federation,  Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Haemostatic efficacy at time of non-surgical bleeding (NSB) event		From Day 1 until final study visit
Primary	Haemostatic efficacy overall		Monthly (prophylactic therapy) or once every 3 months (for on-demand use)
Primary	Number of treatments with blood product transfusions required to resolve any bleeding event		From Day 1 until final study visit
Primary	vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year)		From Day 1 until final study visit
Primary	Assessment of blood loss during any surgical procedure		From Day 1 until final study visit
Primary	Number of spontaneous or traumatic NSB events		From Day 1 until final study visit
Primary	Pharmacokinetic parameters for vWF and FVIII (PK arm only)		Up to 72 hours following infusions on Day 1 and approximately Day 180
Secondary	Development of FVIII inhibitors		From Day 1 until final study visit
Secondary	Development of vWF inhibitors		From Day 1 until final study visit

External Links

•   Click here to request more information about this study