Extension Study of Biostate in Subjects With Von Willebrand Disease - Full Text View

Purpose

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	Intervention	Phase
Von Willebrand Disease	Biological: Biostate	Phase 3

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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

Resource links provided by NLM:

Genetics Home Reference related topics: von Willebrand disease

U.S. FDA Resources

Further study details as provided by CSL Behring:

Primary Outcome Measures:

Haemostatic efficacy [ Time Frame: Up to 32 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Development of FVIII inhibitors [ Time Frame: Up to 32 months ] [ Designated as safety issue: Yes ]
Development of VWF inhibitors [ Time Frame: Up to 32 months ] [ Designated as safety issue: Yes ]
Frequency of Adverse events (AEs) per subject [ Time Frame: 32 months ] [ Designated as safety issue: Yes ]
Severity of AEs per subject [ Time Frame: 32 months ] [ Designated as safety issue: Yes ]
Severity of AEs per infusion [ Time Frame: 32 months ] [ Designated as safety issue: Yes ]
Causality of AEs per subject [ Time Frame: 32 months ] [ Designated as safety issue: Yes ]
Causality of AEs per infusion [ Time Frame: 32 months ] [ Designated as safety issue: Yes ]
Frequency of Adverse events (AEs) per infusion [ Time Frame: 32 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	19
Study Start Date:	October 2010
Estimated Study Completion Date:	May 2014

Arms	Assigned Interventions
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.

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01224808

Contacts

Contact: Clinical Trial Registration Coordinator

clinicaltrials@cslbehring.com

Locations

Bulgaria
Study Site	Active, not recruiting
Sofia, Bulgaria
Germany
Study Site	Recruiting
Bremen, Germany
Poland
Study Site	Active, not recruiting
Warsaw, Poland
Study Site	Active, not recruiting
Wroclaw, Poland
Russian Federation
Study Site	Active, not recruiting
Barnaul, Russian Federation
Ukraine
Study Site	Completed
Lviv, Ukraine

Sponsors and Collaborators

CSL Behring

Investigators

Study Director:

Program Director, Clinical R&D

CSL Behring

More Information

Additional Information:

Click here to request more information about this study This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	CSL Behring
ClinicalTrials.gov Identifier:	NCT01224808 History of Changes
Other Study ID Numbers:	CSLCT-BIO-09-64, 2009-017301-11, 1498
Study First Received:	June 8, 2010
Last Updated:	May 13, 2013
Health Authority:	Bulgaria: Bulgarian Drug Agency Germany: Paul-Ehrlich-Institut Poland: The Central Register of Clinical Trials Russia: Ministry of Health of the Russian Federation Ukraine: State Pharmacological Center - Ministry of Health

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

ClinicalTrials.gov processed this record on May 16, 2013