Von Willebrand Disease MedDRA version: 18.1 Level: PT Classification code 10047715 Term: Von Willebrand's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Voncento
Product Name: Voncento
Pharmaceutical Form: Powder and solvent for solution for injection/infusion
INN or Proposed INN: Human Coagulation Factor VIII
Other descriptive name: HUMAN
Sponsors
CSL Behring GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects meeting all of the following inclusion criteria may be enrolled into the study: 1.Capable of providing written informed consent and willing and able to adhere to all protocol requirements, or the subject’s parent(s) or legally acceptable representative(s) is / are capable of providing written informed consent. 2.Male or female. 3.Subjects of any age. 4.Diagnosis of severe Type 1, 2A, or 3 VWD where VWF:RCo is =65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: Subjects meeting any of the following exclusion criteria must not be enrolled into the study: 1. A known history of VWF or FVIII inhibitors, or are suspected to have VWF or FVIII inhibitors. 2. Suffering any acute or chronic medical condition, other than VWD, which may, in the opinion of the investigator, affect the conduct of the study. 3. Known or suspected hypersensitivity or previous evidence of severe side effects to Voncento, VWF / FVIII concentrates, or human albumin. 4. Participated in another interventional clinical study within 30 days before the first administration of Voncento or at any time during the study. 5. Female subjects who are pregnant, breast-feeding, or who have a positive pregnancy test at screening or have the intention to become pregnant during the course of the study. 6. Female subjects of childbearing potential or male subjects who have a female partner of childbearing potential, who either do not use or are not willing to use a medically reliable method of contraception or who are not sexually abstinent during the study, or not surgically sterile. 7. Alcohol, drug, or medication abuse within 1 year before the study. 8. Currently receiving a therapy not permitted during the study 9. Previous participation in a Voncento / Biostate study (patients can also not be enrolled a second time into the current study). 10. Mental condition rendering the subject (or the subject’s legally acceptable representative[s]) unable to understand the nature, scope, and possible consequences of the study. 11. Any issue that, in the opinion of the investigator, would render the subject unsuitable for participation in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to collect long-term data on the haemostatic efficacy of Voncento in subjects with VWD who require a VWF product to control an NSB event or as prophylaxis therapy.;Secondary Objective: The secondary objectives of the study are: • To assess the efficacy of Voncento in the prevention and treatment of surgical bleeding events. • To collect long-term data on the safety of Voncento used as on-demand therapy to treat an NSB event or as prophylaxis therapy.;Primary end point(s): The primary endpoints of the study are: • Subject’s and investigator’s subjective assessment of haemostatic efficacy of Voncento in its usage for an NSB event. •Number of NSB events per month and annual bleeding rate (ABR). •Number of infusions and total dose of Voncento (in IU VWF:RCo) required to treat an NSB event. ;Timepoint(s) of evaluation of this end point: • Subject’s and investigator’s subjective assessment of haemostatic efficacy of Voncento in its usage for an NSB event: Assessed daily by the subject until the bleed stops, for the duration of the subject's participation in the study (approximately 12 months). Assessed by the Investigator at approximately Months 3, 6, 9 and 12. • Number of infusions: For the duration of the subject's participation in the study (approximately 12 months) •Total dose of Voncento (in IU VWF:RCo) required to treat an NSB event: For the duration of the subject's participation in the study (approximately 12 months). •Number of NSB events per month: For the duration of the subject's participation in the study (approximately 12 months). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoints of the study are: •Subject’s and investigator’s subjective assessment of haemostatic efficacy of Voncento as prophylaxis therapy. •Investigator’s or surgeon’s assessment of blood loss during a surgical event and an overall haemostatic efficacy assessment by the investigator after the surgical procedure. •Number of infusions and total dose of Voncento (in IU VWF:RCo) required to treat a surgical bleeding event. •Nature and incidence of adverse events (AEs), including serious adverse events (SAEs), AEs related to Voncento, and adverse events of special interest (AESIs), and number of VWF and FVIII inhibitors which occur during the administration of Voncento as on-demand therapy to treat an NSB or surgical bleeding event, or as prophylaxis therapy. ;Timepoint(s) of evaluation of this end point: The secondary endpoints of the study are: • Investigator's or surgeon's assessment of blood loss during a surgical event and an overall haemostatic efficacy assessment by the investigator after the surgical procedure. • Number of infusions and total dose of Voncento (in IU VWF:RCo) required to treat a surgical bleeding event. • Nature and incidence of AEs, including serious adverse events (SAEs), AEs related to Voncento, and adverse events of special interest (AESIs), and number of VWF and FVIII inhibitors which occur during the administration of Voncento as on-demand therapy to treat an NSB or surgical bleeding event. •Haemostatic efficacy - prophylaxis: App. every month (subject assessment) and every 3 months (Investigator assessment)during subject's participation in study | — |
Countries
Austria, Germany, Greece, Ireland, Poland, United Kingdom
Contacts
Public ContactKerstin Jung,Clinical Study Manager
CSL Behring GmbH
Outcome results
None listed