Study of Voncento® in Subjects With Von Willebrand Disease

NCT02552576 | Completed Phase 4

• 2 other identifiers: CSLCT-BIO-12-832013-003305-25
• Study Type: interventional
• Target: 26
• Locations: 5 countries
• Sites: 12

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.

Conditions

Von Willebrand Disease

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (7)

• 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

  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

  For the duration of the subject's participation in the study (approximately 12 months).

• Total dose of Voncento - surgical bleeding event

  For the duration of the subject's participation in the study (approximately 12 months).

• Overall adverse events

  From Day 1 until the final study visit for each subject (approximately 12 months)

Study Arms (1)

Voncento

EXPERIMENTAL

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

Interventions

Voncento BIOLOGICAL

Human coagulation VWF / coagulation factor VIII (FVIII) complex concentrate

Voncento

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (12)

Study Site

Vienna, Austria

Location

Study Site

Duisburg, Germany

Location

Study Site

Frankfurt, Germany

Location

Study Site

Athens, Greece

Location

Study Site

Krakow, Poland

Location

Study Site

Rzeszów, Poland

Location

Study Site

Wroclaw, Poland

Location

Study Site 14

London, United Kingdom

Location

Study Site 40

London, United Kingdom

Location

Study Site 42

London, United Kingdom

Location

Study Site 47

London, United Kingdom

Location

Study Site 8

London, United Kingdom

Location

MeSH Terms

Conditions

von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Study Director

  CSL Behring

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 15, 2015
• First Posted: September 17, 2015
• Study Start: October 5, 2015
• Primary Completion: February 15, 2018
• Study Completion: February 15, 2018
• Last Updated: May 16, 2018

Record last verified: 2018-05

Locations

AU (1); GR (1); PL (3); DE (2); GB (5)