Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease
Primary Purpose
Von Willebrand Disease
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Biostate®
Biostate®
Sponsored by
About this trial
This is an interventional treatment trial for Von Willebrand Disease focused on measuring Von Willebrand Disease
Eligibility 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
Sites / Locations
- Study Site
- Study Site
- Study Site
- Study Site
- Study Site
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Experimental
Arm Label
PK
Prophylaxis
On-demand
Cross-over to prophylaxis
Arm Description
Includes subjects participating in the pharmacokinetic component of the study.
Includes subjects receiving 12 months of prophylactic therapy.
Includes subjects receiving 12 months of on-demand treatment.
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.
Outcomes
Primary Outcome Measures
Haemostatic efficacy at time of non-surgical bleeding (NSB) event
Haemostatic efficacy overall
Number of treatments with blood product transfusions required to resolve any bleeding event
vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year)
Assessment of blood loss during any surgical procedure
Number of spontaneous or traumatic NSB events
Pharmacokinetic parameters for vWF and FVIII (PK arm only)
Secondary Outcome Measures
Development of FVIII inhibitors
Development of vWF inhibitors
Full Information
NCT ID
NCT00941616
First Posted
July 15, 2009
Last Updated
October 2, 2017
Sponsor
CSL Behring
Collaborators
Parexel
1. Study Identification
Unique Protocol Identification Number
NCT00941616
Brief Title
Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease
Official Title
An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease.
Study Type
Interventional
2. Study Status
Record Verification Date
October 2017
Overall Recruitment Status
Completed
Study Start Date
June 2009 (undefined)
Primary Completion Date
February 2012 (Actual)
Study Completion Date
February 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CSL Behring
Collaborators
Parexel
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Von Willebrand Disease
Keywords
Von Willebrand Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
22 (Actual)
8. Arms, Groups, and Interventions
Arm Title
PK
Arm Type
Experimental
Arm Description
Includes subjects participating in the pharmacokinetic component of the study.
Arm Title
Prophylaxis
Arm Type
Experimental
Arm Description
Includes subjects receiving 12 months of prophylactic therapy.
Arm Title
On-demand
Arm Type
Experimental
Arm Description
Includes subjects receiving 12 months of on-demand treatment.
Arm Title
Cross-over to prophylaxis
Arm Type
Experimental
Arm Description
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 Type
Biological
Intervention Name(s)
Biostate®
Other Intervention Name(s)
Human Coagulation Factor VIII / von Willebrand Factor
Intervention Description
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Intervention Type
Biological
Intervention Name(s)
Biostate®
Other Intervention Name(s)
Human Coagulation Factor VIII / von Willebrand Factor
Intervention Description
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 Measure Information:
Title
Haemostatic efficacy at time of non-surgical bleeding (NSB) event
Time Frame
From Day 1 until final study visit
Title
Haemostatic efficacy overall
Time Frame
Monthly (prophylactic therapy) or once every 3 months (for on-demand use)
Title
Number of treatments with blood product transfusions required to resolve any bleeding event
Time Frame
From Day 1 until final study visit
Title
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
Title
Assessment of blood loss during any surgical procedure
Time Frame
From Day 1 until final study visit
Title
Number of spontaneous or traumatic NSB events
Time Frame
From Day 1 until final study visit
Title
Pharmacokinetic parameters for vWF and FVIII (PK arm only)
Time Frame
Up to 72 hours following infusions on Day 1 and approximately Day 180
Secondary Outcome Measure Information:
Title
Development of FVIII inhibitors
Time Frame
From Day 1 until final study visit
Title
Development of vWF inhibitors
Time Frame
From Day 1 until final study visit
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Program Director, Clinical R&D
Organizational Affiliation
CSL Behring
Official's Role
Study Director
Facility Information:
Facility Name
Study Site
City
Sofia
Country
Bulgaria
Facility Name
Study Site
City
Warsaw
Country
Poland
Facility Name
Study Site
City
Wroclaw
Country
Poland
Facility Name
Study Site
City
Barnaul
Country
Russian Federation
Facility Name
Study Site
City
Lviv
ZIP/Postal Code
79044
Country
Ukraine
12. IPD Sharing Statement
Links:
URL
http://www.cslbehring.com/clinical-trials/contact-us.htm?registryRefNum=NCT00941616®istryName=ctgov
Description
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Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease
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