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

This study has been completed.
Sponsor:
Collaborator:
Parexel
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT00941616
First received: July 15, 2009
Last updated: April 4, 2013
Last verified: April 2013

July 15, 2009
April 4, 2013
June 2009
Not Provided
  • Haemostatic efficacy at time of non-surgical bleeding (NSB) event [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
  • Haemostatic efficacy overall [ Time Frame: Monthly (prophylactic therapy) or once every 3 months (for on-demand use) ] [ Designated as safety issue: No ]
  • Number of treatments with blood product transfusions required to resolve any bleeding event [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Assessment of blood loss during any surgical procedure [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
  • Number of spontaneous or traumatic NSB events [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
  • Pharmacokinetic parameters for vWF and FVIII (PK arm only) [ Time Frame: Up to 72 hours following infusions on Day 1 and approximately Day 180 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: Yes ]
  • Development of vWF inhibitors [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: Yes ]
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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: 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
Not Provided

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
Both
12 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Bulgaria,   Poland,   Russian Federation,   Ukraine
 
NCT00941616
CSLCT-BIO-08-54, 1481, 2008-004922-18
Yes
CSL Behring
CSL Behring
Parexel
Study Director: Program Director, Clinical R&D CSL Behring
CSL Behring
April 2013

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