Extension Study of Biostate in Subjects With Von Willebrand Disease

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. Identifier: NCT01224808
Recruitment Status : Completed
First Posted : October 20, 2010
Last Update Posted : October 3, 2017
Information provided by (Responsible Party):
CSL Behring

Brief Summary:
The aim of the Von Willebrand Disease (VWD) therapy is to treat and prevent bleeding episodes due to abnormal platelet adhesion and abnormal blood coagulation as a result of low or abnormal Von Willebrand Factor (VWF) and/or Factor VIII (FVIII) levels. The long-term efficacy and safety of a VWF/FVIII concentrate, Biostate, will be investigated in children, adolescents, and adults with VWD in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.

Condition or disease Intervention/treatment Phase
Von Willebrand Disease Biological: Biostate Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Multi-Centre Extension Study to Assess the Efficacy and Safety of Biostate® in Paediatric, Adolescent, and Adult Subjects With Von Willebrand Disease Who Completed Clinical Studies CSLCT-BIO-08-52 or CSLCTBIO-08-54
Study Start Date : October 2010
Actual Primary Completion Date : March 2014
Actual Study Completion Date : March 2014

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: Experimental Biological: Biostate
Single bolus doses, administered intravenously. 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.

Primary Outcome Measures :
  1. Haemostatic efficacy [ Time Frame: Up to 32 months ]

Secondary Outcome Measures :
  1. Development of FVIII inhibitors [ Time Frame: Up to 32 months ]
  2. Development of VWF inhibitors [ Time Frame: Up to 32 months ]
  3. Frequency of Adverse events (AEs) per subject [ Time Frame: 32 months ]
  4. Severity of AEs per subject [ Time Frame: 32 months ]
  5. Severity of AEs per infusion [ Time Frame: 32 months ]
  6. Causality of AEs per subject [ Time Frame: 32 months ]
  7. Causality of AEs per infusion [ Time Frame: 32 months ]
  8. Frequency of Adverse events (AEs) per infusion [ Time Frame: 32 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Have completed Study CSLCT-BIO-08-52 (Assessment of Efficacy and Safety of Biostate in Paediatric Subjects with Von Willebrand Disease) or Study CSLCT-BIO-08-54 (Assessment of Efficacy and Safety of Biostate in Adolescent or Adult Subjects with Von Willebrand Disease).
  • The subject and/or his/her legal guardian understand(s) the nature of the study and has/have given written informed consent to participate in the study and is/are willing to comply with the protocol.

Exclusion Criteria:

  • Early discontinuation of a subject from the main studies CSLCT-BIO-08-52 or CSLCT-BIO-08-54.
  • Mental condition rendering the subject (or the subject's legal guardian) unable to understand the nature, scope and possible consequences of the study.
  • Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study.
  • Are not willing and/or not able to comply with the study requirements.
  • Employee at the study site, or spouse/partner or relative of the Investigator or Subinvestigators.
  • Female subjects of childbearing potential either not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study, or not sexually abstinent for the entire duration of the study, or not surgically sterile.
  • Intention to become pregnant during the course of the study.
  • Pregnancy, or nursing mother.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01224808

Study Site
Sofia, Bulgaria
Study Site
Bremen, Germany
Study Site
Warsaw, Poland
Study Site
Wroclaw, Poland
Russian Federation
Study Site
Barnaul, Russian Federation
Study Site
Lviv, Ukraine
Sponsors and Collaborators
CSL Behring
Study Director: Program Director, Clinical R&D CSL Behring

Additional Information:
Responsible Party: CSL Behring Identifier: NCT01224808     History of Changes
Other Study ID Numbers: CSLCT-BIO-09-64
2009-017301-11 ( EudraCT Number )
1498 ( Other Identifier: CSL Behring )
First Posted: October 20, 2010    Key Record Dates
Last Update Posted: October 3, 2017
Last Verified: October 2017

Keywords provided by CSL Behring:
von Willebrand Disease

Additional relevant MeSH terms:
Von Willebrand Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn