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Study of Voncento® in Subjects With Von Willebrand Disease

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

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 26 participants
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

Resource links provided by the National Library of Medicine


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




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.



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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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02552576


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

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT02552576     History of Changes
Other Study ID Numbers: CSLCT-BIO-12-83
2013-003305-25 ( EudraCT Number )
First Posted: September 17, 2015    Key Record Dates
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