Clinical trial NCT02552576: Study of Voncento® in Subjects With Von Willebrand Disease
Official title: An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects With Von Willebrand Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Von Willebrand Disease
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2015-09-17
Enrollment: 26
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Haemostatic efficacy - NSB event
timeFrame: 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.
measure: Number of infusions - NSB event
timeFrame: For the duration of the subject's participation in the study (approximately 12 months).
measure: Total dose of Voncento - NSB event
timeFrame: For the duration of the subject's participation in the study (approximately 12 months).
measure: Number of NSB events per month
timeFrame: From Day 1 until final study visit, approximately 12 months.
measure: Annual bleeding rate
timeFrame: For the duration of the subject's participation in the study (approximately 12 months).
Secondary outcomes:
measure: Assessment of blood loss during a surgical procedure
timeFrame: During surgery, for any surgical procedure during the subject's participation in the study (approximately 12 months).
measure: Haemostatic efficacy - surgical event
timeFrame: Assessed during and after surgery until the bleeding stops, for any surgical procedure during the subject's participation in the study (approximately 12 months).
measure: Number of infusions - surgical bleeding event
timeFrame: For the duration of the subject's participation in the study (approximately 12 months).
measure: Total dose of Voncento - surgical bleeding event
timeFrame: For the duration of the subject's participation in the study (approximately 12 months).
measure: Overall adverse events
timeFrame: From Day 1 until the final study visit for each subject (approximately 12 months)
measure: Number of subjects with VWF or FVIII inhibitors
timeFrame: At screening, Day 1 and approximately Months 3, 6, 9 and 12, for each subject.
measure: Haemostatic efficacy - prophylaxis
timeFrame: 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).
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Study Site
city: Duisburg
country: Germany
facility: Study Site
city: Frankfurt
country: Germany
facility: Study Site
city: Athens
country: Greece
facility: Study Site
city: Krakow
country: Poland
facility: Study Site
city: Rzeszów
country: Poland
facility: Study Site
city: Wroclaw
country: Poland
facility: Study Site
city: London
country: United Kingdom
facility: Study Site 14
city: London
country: United Kingdom
facility: Study Site 40
city: London
country: United Kingdom
facility: Study Site 42
city: London
country: United Kingdom
facility: Study Site 47
city: London
country: United Kingdom
facility: Study Site 8
Study officials:
affiliation: CSL Behring
name: Study Director
role: STUDY_DIRECTOR
References: