Study of Voncento® in Subjects With Von Willebrand Disease
Primary Purpose
Von Willebrand Disease
Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Voncento
Sponsored by
About this trial
This is an interventional treatment trial for Von Willebrand Disease
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of severe type 1, 2A, or 3 VWD where Von Willebrand Factor: Ristocetin Cofactor (VWF:RCo) is <20% at screening
- Desmopressin acetate treatment is ineffective, contraindicated, or not available for subject (type 3 VWD subjects only).
- Evidence of vaccination against hepatitis A and B or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunization.
- Written informed consent given.
- Require a VWF product to control a non-surgical bleeding (NSB) event or for ongoing prophylactic therapy.
Exclusion Criteria:
- Known history or suspicion of having VWF or FVIII inhibitors
- 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 Voncento, VWF / FVIII concentrates, or human albumin.
- Participated in another interventional clinical study within 30 days before the first administration of Voncento or at any time during the study.
- Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening
- Alcohol, drug, or medication abuse within 1 year before the study.
- Currently receiving a therapy not permitted during the study.
- Previous participation in a Voncento / Biostate study.
Sites / Locations
- Study Site
- Study Site
- Study Site
- Study Site
- Study Site
- Study Site
- Study Site
- Study Site 14
- Study Site 40
- Study Site 42
- Study Site 47
- Study Site 8
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Voncento
Arm Description
The frequency and dose of Voncento administration will be determined by the investigator using the information included in the Voncento Summary of product characteristics (SmPC)
Outcomes
Primary Outcome Measures
Haemostatic efficacy - NSB event
Subject's and investigator's assessment of haemostatic efficacy of Voncento in its usage for a non-surgical bleeding (NSB) event. Assessments of haemostatic efficacy will be based on a 4-point ordinal scale of Excellent, Good, Moderate or None.
Number of infusions - NSB event
Number of infusions of Voncento required to treat an NSB event.
Total dose of Voncento - NSB event
Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat an NSB event.
Number of NSB events per month
Annual bleeding rate
The number of bleeding events per year
Secondary Outcome Measures
Assessment of blood loss during a surgical procedure
Haemostatic efficacy - surgical event
Investigator's or surgeon's assessment of haemostatic efficacy of Voncento in its usage for a surgical bleeding event. Assessments of haemostatic efficacy will be based on a 4-point ordinal scale of Excellent, Good, Moderate or None.
Number of infusions - surgical bleeding event
Number of infusions of Voncento required to treat a surgical bleeding event.
Total dose of Voncento - surgical bleeding event
Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat a surgical bleeding event.
Overall adverse events
Overall number of subjects with: adverse events (AEs), serious AEs, AEs related to Voncento administration, and adverse events of special interest.
Number of subjects with VWF or FVIII inhibitors
Haemostatic efficacy - prophylaxis
Subject's and investigator's assessment of haemostatic efficacy of Voncento as prophylaxis therapy. Assessments of haemostatic efficacy will be based on a grading scale with outcomes of excellent, good, moderate, none.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02552576
Brief Title
Study of Voncento® in Subjects With Von Willebrand Disease
Official Title
An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects With Von Willebrand Disease
Study Type
Interventional
2. Study Status
Record Verification Date
May 2018
Overall Recruitment Status
Completed
Study Start Date
October 5, 2015 (Actual)
Primary Completion Date
February 15, 2018 (Actual)
Study Completion Date
February 15, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CSL Behring
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a multi-centre, open-label, single-arm, phase 4, post-marketing study to further investigate the efficacy and safety of Voncento in subjects with Von Willebrand Disease (VWD) in whom treatment with a Von Willebrand Factor (VWF) product is required as on-demand therapy, for prophylactic therapy, or during surgery. Subjects will be treated with Voncento either as an on-demand regimen (eg, to treat a non-surgical spontaneous or traumatic bleeding event) or prevention regimen (eg, to prevent an anticipated bleeding event) at a dose prescribed by the Investigator in accordance with the Voncento Summary of Product Characteristics (SmPC), or with a prophylaxis regimen (regular treatment with Voncento at a frequency of 1-3 times per week). Voncento will also be given to prevent and treat any surgical bleeding events.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Von Willebrand Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
26 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Voncento
Arm Type
Experimental
Arm Description
The frequency and dose of Voncento administration will be determined by the investigator using the information included in the Voncento Summary of product characteristics (SmPC)
Intervention Type
Biological
Intervention Name(s)
Voncento
Intervention Description
Human coagulation VWF / coagulation factor VIII (FVIII) complex concentrate
Primary Outcome Measure Information:
Title
Haemostatic efficacy - NSB event
Description
Subject's and investigator's assessment of haemostatic efficacy of Voncento in its usage for a non-surgical bleeding (NSB) event. Assessments of haemostatic efficacy will be based on a 4-point ordinal scale of Excellent, Good, Moderate or None.
Time Frame
Assessed daily by the subject until the bleed stops, for the duration of the subject's participation in the study (approximately 12 months). Each bleeding event is also to be assessed retrospectively by the Investigator.
Title
Number of infusions - NSB event
Description
Number of infusions of Voncento required to treat an NSB event.
Time Frame
For the duration of the subject's participation in the study (approximately 12 months).
Title
Total dose of Voncento - NSB event
Description
Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat an NSB event.
Time Frame
For the duration of the subject's participation in the study (approximately 12 months).
Title
Number of NSB events per month
Time Frame
From Day 1 until final study visit, approximately 12 months.
Title
Annual bleeding rate
Description
The number of bleeding events per year
Time Frame
For the duration of the subject's participation in the study (approximately 12 months).
Secondary Outcome Measure Information:
Title
Assessment of blood loss during a surgical procedure
Time Frame
During surgery, for any surgical procedure during the subject's participation in the study (approximately 12 months).
Title
Haemostatic efficacy - surgical event
Description
Investigator's or surgeon's assessment of haemostatic efficacy of Voncento in its usage for a surgical bleeding event. Assessments of haemostatic efficacy will be based on a 4-point ordinal scale of Excellent, Good, Moderate or None.
Time Frame
Assessed during and after surgery until the bleeding stops, for any surgical procedure during the subject's participation in the study (approximately 12 months).
Title
Number of infusions - surgical bleeding event
Description
Number of infusions of Voncento required to treat a surgical bleeding event.
Time Frame
For the duration of the subject's participation in the study (approximately 12 months).
Title
Total dose of Voncento - surgical bleeding event
Description
Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat a surgical bleeding event.
Time Frame
For the duration of the subject's participation in the study (approximately 12 months).
Title
Overall adverse events
Description
Overall number of subjects with: adverse events (AEs), serious AEs, AEs related to Voncento administration, and adverse events of special interest.
Time Frame
From Day 1 until the final study visit for each subject (approximately 12 months)
Title
Number of subjects with VWF or FVIII inhibitors
Time Frame
At screening, Day 1 and approximately Months 3, 6, 9 and 12, for each subject.
Title
Haemostatic efficacy - prophylaxis
Description
Subject's and investigator's assessment of haemostatic efficacy of Voncento as prophylaxis therapy. Assessments of haemostatic efficacy will be based on a grading scale with outcomes of excellent, good, moderate, none.
Time Frame
Approximately every month (subject assessment) and every 3 months (Investigator assessment) for the duration of the subject's participation in the study (approximately 12 months).
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of severe type 1, 2A, or 3 VWD where Von Willebrand Factor: Ristocetin Cofactor (VWF:RCo) is <20% at screening
Desmopressin acetate treatment is ineffective, contraindicated, or not available for subject (type 3 VWD subjects only).
Evidence of vaccination against hepatitis A and B or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunization.
Written informed consent given.
Require a VWF product to control a non-surgical bleeding (NSB) event or for ongoing prophylactic therapy.
Exclusion Criteria:
Known history or suspicion of having VWF or FVIII inhibitors
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 Voncento, VWF / FVIII concentrates, or human albumin.
Participated in another interventional clinical study within 30 days before the first administration of Voncento or at any time during the study.
Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening
Alcohol, drug, or medication abuse within 1 year before the study.
Currently receiving a therapy not permitted during the study.
Previous participation in a Voncento / Biostate study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
CSL Behring
Official's Role
Study Director
Facility Information:
Facility Name
Study Site
City
Vienna
Country
Austria
Facility Name
Study Site
City
Duisburg
Country
Germany
Facility Name
Study Site
City
Frankfurt
Country
Germany
Facility Name
Study Site
City
Athens
Country
Greece
Facility Name
Study Site
City
Krakow
Country
Poland
Facility Name
Study Site
City
Rzeszów
Country
Poland
Facility Name
Study Site
City
Wroclaw
Country
Poland
Facility Name
Study Site 14
City
London
Country
United Kingdom
Facility Name
Study Site 40
City
London
Country
United Kingdom
Facility Name
Study Site 42
City
London
Country
United Kingdom
Facility Name
Study Site 47
City
London
Country
United Kingdom
Facility Name
Study Site 8
City
London
Country
United Kingdom
12. IPD Sharing Statement
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Study of Voncento® in Subjects With Von Willebrand Disease
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