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

October 2, 2017 updated by: CSL Behring

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

22

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Bulgaria
      • Sofia, Bulgaria
        • Study Site
  • Poland
      • Warsaw, Poland
        • Study Site
      • Wroclaw, Poland
        • Study Site
  • Russian Federation
      • Barnaul, Russian Federation
        • Study Site
  • Ukraine
      • Lviv, Ukraine, 79044
        • Study Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: PK
Includes subjects participating in the pharmacokinetic component of the study.
Biological: Biostate®
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
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.
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
Experimental: Prophylaxis
Includes subjects receiving 12 months of prophylactic therapy.
Biological: Biostate®
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
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.
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
Experimental: On-demand
Includes subjects receiving 12 months of on-demand treatment.
Biological: Biostate®
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
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.
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
Experimental: Cross-over to prophylaxis
Includes subjects completing 12 months of on-demand treatment (the "On-demand" arm) who cross-over to prophylactic therapy for an additional 12-month period.
Biological: Biostate®
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor
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.
Other Names:
  • Human Coagulation Factor VIII / von Willebrand Factor

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Development of FVIII inhibitors
Time Frame: From Day 1 until final study visit
From Day 1 until final study visit
Development of vWF inhibitors
Time Frame: From Day 1 until final study visit
From Day 1 until final study visit

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CSL Behring

Collaborators

Parexel

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2009

Primary Completion (Actual)

February 1, 2012

Study Completion (Actual)

February 1, 2012

Study Registration Dates

First Submitted

July 15, 2009

First Submitted That Met QC Criteria

July 15, 2009

First Posted (Estimate)

July 17, 2009

Study Record Updates

Last Update Posted (Actual)

October 3, 2017

Last Update Submitted That Met QC Criteria

October 2, 2017

Last Verified

October 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CSLCT-BIO-08-54
  • 1481 (Other Identifier: CSL Behring)
  • 2008-004922-18 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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