Study of Voncento® in Subjects With Von Willebrand Disease

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

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.

Study Overview

• Status: Completed
• Conditions: Von Willebrand Disease
• Intervention / Treatment: Biological: Voncento

• Study Type: Interventional
• Enrollment (Actual): 26
• Phase: Phase 4

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria
    • Study Site
• Germany
  • Duisburg, Germany
    • Study Site
  • Frankfurt, Germany
    • Study Site
• Greece
  • Athens, Greece
    • Study Site
• Poland
  • Krakow, Poland
    • Study Site
  • Rzeszów, Poland
    • Study Site
  • Wroclaw, Poland
    • Study Site
• United Kingdom
  • London, 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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Voncento; 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)	Biological: Voncento; Human coagulation VWF / coagulation factor VIII (FVIII) complex concentrate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.
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).
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).
Number of NSB events per month		From Day 1 until final study visit, approximately 12 months.
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).
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).
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).
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).
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)
Number of subjects with VWF or FVIII inhibitors		At screening, Day 1 and approximately Months 3, 6, 9 and 12, for each subject.
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).

Collaborators and Investigators

• Sponsor: CSL Behring

Study record dates

Study Major Dates

• Study Start (ACTUAL): October 5, 2015
• Primary Completion (ACTUAL): February 15, 2018
• Study Completion (ACTUAL): February 15, 2018

Study Registration Dates

• First Submitted: September 15, 2015
• First Submitted That Met QC Criteria: September 16, 2015
• First Posted (ESTIMATE): September 17, 2015

Study Record Updates

• Last Update Posted (ACTUAL): May 16, 2018
• Last Update Submitted That Met QC Criteria: May 15, 2018
• Last Verified: May 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Blood Platelet Disorders; Von Willebrand Diseases
• Other Study ID Numbers: CSLCT-BIO-12-83; 2013-003305-25 (EUDRACT_NUMBER)