Von Willebrand's disease (VWD) MedDRA version: 17.1 Level: PT Classification code 10047715 Term: Von Willebrand's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Voncento, Biostate®
Product Name: Human coagulation Factor VIII / von Willebrand Factor
Pharmaceutical Form: Powder and solvent for solution for injection/infusion
Sponsors
CSL Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients who fulfill the following criteria are eligible for inclusion into this study: a) Patients with a diagnosis VWD who require prophylactic therapy, have a non-surgery bleed, or are undergoing surgery. b) Patients who would routinely receive a plasma derived FVIII/VWF product as treatment for their VWD. c) Patients over 3 years of age and =14 kg. Inclusion of children =65 years) no F.1.3.1 Number of subjects for this age range 13
Exclusion criteria
Exclusion criteria: The following patients are not eligible for inclusion into this study: a) Patients with a known history of adverse drug reactions to factor VIII/VWF concentrates. b) Patients with a known or suspected VWF or FVIII inhibitor. c) Patients who are currently taking non-steroidal anti-inflammatory drugs (NSAIDs) which in the opinion of the Investigator would affect the efficacy and safety outcomes of the study. d) Patients with evidence or a history within the previous 12 months of abuse of any drug substance, licit or illicit, which in the opinion of the Investigator may affect the ability of the patient to comply with all protocol requirements. e) Patients who have within 90 days prior to the study screening visit, participated in a clinical study or used an investigational compound (eg. a new chemical entity not registered for clinical use), or who are planning to enter such a different study during the study period. f) Patients who are suffering from an acute or chronic medical condition, other than VWD, which may, in the opinion of the Investigator, affect the conduct of the study. g) Patients with a positive pregnancy test.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objectives of this study are to evaluate the: • Efficacy and • Safety of Biostate® in the treatment of non-surgery bleeds, in the management of surgery procedures and prophylactic therapy in patients with VWD where 1-deamino-8-D-arginine vasopressin/Desmopressin (DDAVP) treatment is deemed by the Investigator to be ineffective, inadequate, or contraindicated. ;Secondary Objective: Not applicable;Primary end point(s): Efficacy Endpoints: • Assessment of haemostatic efficacy using a grading scale of excellent, good, moderate and none. • For non-surgery bleed/surgery procedures: - The plasma levels of FVIII:C, VWF:RCo, VWF:Ag and VWF:CB. - Blood transfusion requirements. - FVIII:C and VWF:RCo in IU/kg (per treatment) required to resolve the event. - The number of treatments required until resolution of the event. - Assessment of blood loss during any surgery procedures. • For prophylaxis therapy: - FVIII:C IU/kg and VWF:RCo IU/kg per month. - Number of spontaneous bleeding events. Safety Endpoints: • The nature and incidence of AEs. • The development of inhibitors to FVIII. • The use of concomitant medications.;Timepoint(s) of evaluation of this end point: Up to 13 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): not applicable;Timepoint(s) of evaluation of this end point: not applicable | — |
Countries
Australia
Contacts
Public ContactTrial Registration Co-ordinator
CSL Behring
Outcome results
None listed