Study of Voncento® in Subjects With Von Willebrand Disease

CSL Behring

Status and phase: Completed; Phase 4

Conditions: Von Willebrand Disease

Treatments: Biological: Voncento

Study type: Interventional

Funder types: Industry

Identifiers

NCT02552576

2013-003305-25 (EudraCT Number)

CSLCT-BIO-12-83

Details and patient eligibility

About

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.

Enrollment: 26 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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26 participants in 1 patient group

Voncento

Experimental group

Description:

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)

Treatment:

Biological: Voncento