Study of a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate (vWF/FVIII), Biostate®, in Subjects With Von Willebrand Disease - Full Text View

Sponsor:	CSL Behring
Collaborator:	Parexel
Information provided by:	CSL Behring
ClinicalTrials.gov Identifier:	NCT00941616

Purpose

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.

Condition	Intervention	Phase
Von Willebrand Disease	Biological: Biostate®	Phase II Phase III

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Parallel Assignment, Safety/Efficacy Study
Official Title:	An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease.

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia von Willebrand disease Help Me Understand Genetics

Drug Information available for: Octocog alfa Moroctocog Alfa Factor VIII

U.S. FDA Resources

Further study details as provided by CSL Behring:

Primary Outcome Measures:

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 ]

Secondary Outcome Measures:

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 ]

Estimated Enrollment:	25
Study Start Date:	June 2009
Estimated Study Completion Date:	November 2011
Estimated Primary Completion Date:	November 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
PK: Experimental Includes subjects participating in the pharmacokinetic component of the study.	Biological: Biostate® 80 IU/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Prophylaxis: Experimental Includes subjects receiving 12 months of prophylactic therapy.	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.
On-demand: Experimental Includes subjects receiving 12 months of on-demand treatment.	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.
Cross-over to prophylaxis: Experimental 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® 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

Ages Eligible for Study:	12 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00941616

Contacts

Contact: Central Contact: Clinical Trial Registration Coordinator

clinicaltrials@cslbehring.com

Locations

Brazil
Study Site	Not yet recruiting
Rio de Janeiro, Brazil
Contact: Use Central Contact
Bulgaria
Study Site	Recruiting
Sofia, Bulgaria
Contact: Use Central Contact
Poland
Study Site	Recruiting
Warsaw, Poland
Contact: Use Central Contact
Study Site	Recruiting
Wroclaw, Poland
Contact: Use Central Contact
Russian Federation
Study Site	Recruiting
Barnaul, Russian Federation
Contact: Use Central Contact
Ukraine
Study Site	Recruiting
Lviv, Ukraine, 79044
Contact: Use Central Contact

Sponsors and Collaborators

CSL Behring

Parexel

Investigators

Study Director:

Program Director, Clinical R&D

CSL Behring

More Information

No publications provided

Responsible Party:	CSL Behring ( Global Head Clinical Research & Development )
Study ID Numbers:	1481, CSLCT-BIO-08-54, 2008-004922-18
Study First Received:	July 15, 2009
Last Updated:	January 14, 2010
ClinicalTrials.gov Identifier:	NCT00941616 History of Changes
Health Authority:	Russia: Ministry of Health and Social Development of the Russian Federation; Bulgaria: Bulgarian Drug Agency; Brazil: National Health Surveillance Agency; Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products; Ukraine: State Pharmacological Center - Ministry of Health

Keywords provided by CSL Behring:

Von Willebrand Disease

Additional relevant MeSH terms:

Von Willebrand Disease
Coagulants
Hematologic Diseases
Coagulation Protein Disorders
Blood Coagulation Disorders
Blood Platelet Disorders
Hematologic Agents

Factor VIII
Pharmacologic Actions
Blood Coagulation Disorders, Inherited
Hemorrhagic Disorders
Genetic Diseases, Inborn
Therapeutic Uses

ClinicalTrials.gov processed this record on February 08, 2010