Study of Voncento® in Subjects With Von Willebrand Disease

Von Willebrand Disease Clinical Trial

Official title:

An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects With Von Willebrand Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02552576
• Other study ID #: CSLCT-BIO-12-83
• Secondary ID: 2013-003305-25
• Status: Completed
• Phase: Phase 4
• Start date: October 5, 2015
• Est. completion date: February 15, 2018

Study information

• Verified date: May 2018
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 26
• Est. completion date: February 15, 2018
• Est. primary completion date: February 15, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Von Willebrand Disease
• Von Willebrand Diseases

Intervention

Biological:
• Voncento
Human coagulation VWF / coagulation factor VIII (FVIII) complex concentrate

Locations

Country	Name	City	State
Austria	Study Site	Vienna
Germany	Study Site	Duisburg
Germany	Study Site	Frankfurt
Greece	Study Site	Athens
Poland	Study Site	Krakow
Poland	Study Site	Rzeszów
Poland	Study Site	Wroclaw
United Kingdom	Study Site 14	London
United Kingdom	Study Site 40	London
United Kingdom	Study Site 42	London
United Kingdom	Study Site 47	London
United Kingdom	Study Site 8	London

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

Austria,  Germany,  Greece,  Poland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	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.
Primary	Number of infusions - NSB event	Number of infusions of Voncento required to treat an NSB event.	For the duration of the subject's participation in the study (approximately 12 months).
Primary	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.	For the duration of the subject's participation in the study (approximately 12 months).
Primary	Number of NSB events per month		From Day 1 until final study visit, approximately 12 months.
Primary	Annual bleeding rate	The number of bleeding events per year	For the duration of the subject's participation in the study (approximately 12 months).
Secondary	Assessment of blood loss during a surgical procedure		During surgery, for any surgical procedure during the subject's participation in the study (approximately 12 months).
Secondary	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.	Assessed during and after surgery until the bleeding stops, for any surgical procedure during the subject's participation in the study (approximately 12 months).
Secondary	Number of infusions - surgical bleeding event	Number of infusions of Voncento required to treat a surgical bleeding event.	For the duration of the subject's participation in the study (approximately 12 months).
Secondary	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.	For the duration of the subject's participation in the study (approximately 12 months).
Secondary	Overall adverse events	Overall number of subjects with: adverse events (AEs), serious AEs, AEs related to Voncento administration, and adverse events of special interest.	From Day 1 until the final study visit for each subject (approximately 12 months)
Secondary	Number of subjects with VWF or FVIII inhibitors		At screening, Day 1 and approximately Months 3, 6, 9 and 12, for each subject.
Secondary	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.	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).