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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00941616
First Posted: July 17, 2009
Last Update Posted: October 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Parexel
Information provided by (Responsible Party):
CSL Behring
July 15, 2009
July 17, 2009
October 3, 2017
June 2009
February 2012   (Final data collection date for primary outcome measure)
  • Haemostatic efficacy at time of non-surgical bleeding (NSB) event [ Time Frame: From Day 1 until final study visit ]
  • Haemostatic efficacy overall [ Time Frame: 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 ]
  • 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 ]
  • Assessment of blood loss during any surgical procedure [ Time Frame: From Day 1 until final study visit ]
  • Number of spontaneous or traumatic NSB events [ Time Frame: 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 ]
Same as current
Complete list of historical versions of study NCT00941616 on ClinicalTrials.gov Archive Site
  • Development of FVIII inhibitors [ Time Frame: From Day 1 until final study visit ]
  • Development of vWF inhibitors [ Time Frame: From Day 1 until final study visit ]
Same as current
Not Provided
Not Provided
 
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.

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.

Not Provided
Interventional
Phase 2
Phase 3
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Von Willebrand Disease
  • Biological: Biostate®
    80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
    Other Name: 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 Name: Human Coagulation Factor VIII / von Willebrand Factor
  • Experimental: PK
    Includes subjects participating in the pharmacokinetic component of the study.
    Intervention: Biological: Biostate®
  • Experimental: Prophylaxis
    Includes subjects receiving 12 months of prophylactic therapy.
    Intervention: Biological: Biostate®
  • Experimental: On-demand
    Includes subjects receiving 12 months of on-demand treatment.
    Intervention: Biological: Biostate®
  • 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.
    Intervention: Biological: Biostate®
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
22
February 2012
February 2012   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
12 Years and older   (Child, Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Bulgaria,   Poland,   Russian Federation,   Ukraine
Brazil
 
NCT00941616
CSLCT-BIO-08-54
1481 ( Other Identifier: CSL Behring )
2008-004922-18 ( EudraCT Number )
Yes
Not Provided
Not Provided
CSL Behring
CSL Behring
Parexel
Study Director: Program Director, Clinical R&D CSL Behring
CSL Behring
October 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP