Study of Voncento® in Subjects With Von Willebrand Disease

• ClinicalTrials.gov Identifier: NCT02552576
• Recruitment Status: Completed
• First Posted: September 17, 2015
• Last Update Posted: May 16, 2018
• Sponsor: CSL Behring
• Information provided by (Responsible Party): CSL Behring

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.

Condition or disease	Intervention/treatment	Phase
Von Willebrand Disease	Biological: Voncento	Phase 4

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 26 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects With Von Willebrand Disease
• Actual Study Start Date: October 5, 2015
• Actual Primary Completion Date: February 15, 2018
• Actual Study Completion Date: February 15, 2018

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. Haemostatic efficacy - NSB event [ 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. ]
   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.
2. Number of infusions - NSB event [ Time Frame: For the duration of the subject's participation in the study (approximately 12 months). ]
   Number of infusions of Voncento required to treat an NSB event.
3. Total dose of Voncento - NSB event [ Time Frame: For the duration of the subject's participation in the study (approximately 12 months). ]
   Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat an NSB event.
4. Number of NSB events per month [ Time Frame: From Day 1 until final study visit, approximately 12 months. ]
5. Annual bleeding rate [ Time Frame: For the duration of the subject's participation in the study (approximately 12 months). ]
   The number of bleeding events per year

Secondary Outcome Measures :

1. 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). ]
2. Haemostatic efficacy - surgical event [ 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). ]
   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.
3. Number of infusions - surgical bleeding event [ Time Frame: For the duration of the subject's participation in the study (approximately 12 months). ]
   Number of infusions of Voncento required to treat a surgical bleeding event.
4. Total dose of Voncento - surgical bleeding event [ Time Frame: For the duration of the subject's participation in the study (approximately 12 months). ]
   Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat a surgical bleeding event.
5. Overall adverse events [ Time Frame: From Day 1 until the final study visit for each subject (approximately 12 months) ]
   Overall number of subjects with: adverse events (AEs), serious AEs, AEs related to Voncento administration, and adverse events of special interest.
6. 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. ]
7. Haemostatic efficacy - prophylaxis [ 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). ]
   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.

Eligibility Criteria

• Ages Eligible for Study: Child, Adult, Older Adult
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

Austria

Study Site

Vienna, Austria

Germany

Study Site

Duisburg, Germany

Study Site

Frankfurt, Germany

Greece

Study Site

Athens, Greece

Poland

Study Site

Krakow, Poland

Study Site

Rzeszów, Poland

Study Site

Wroclaw, Poland

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, United Kingdom

Sponsors and Collaborators

CSL Behring

Investigators

• Study Director: Study Director; CSL Behring

More Information

• Responsible Party: CSL Behring
• ClinicalTrials.gov Identifier: NCT02552576
• Other Study ID Numbers: CSLCT-BIO-12-83; 2013-003305-25 ( EudraCT Number )
• First Posted: September 17, 2015
• Last Update Posted: May 16, 2018
• Last Verified: May 2018

Additional relevant MeSH terms:

Von Willebrand Diseases; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn