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Completed

NCT Number: NCT02552576

Study 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.

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Key information

Conditions

Von Willebrand Disease Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Blood Platelet Disorders Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders von Willebrand Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Study Site, Vienna, Austria

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Voncento

Biological

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

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

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

Important dates

Study start
2015
Primary completion
2018
Study completion
2018
First posted
Sep 17, 2015
Registry last updated
May 16, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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