Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
MY TRIALS
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Register a trial
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01410227
Registration number
NCT01410227
Ethics application status
Date submitted
4/08/2011
Date registered
5/08/2011
Titles & IDs
Public title
Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment of Bleeding Episodes in Von Willebrand Disease (VWD)
Query!
Scientific title
A Phase 3 Clinical Study to Determine the Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor : Recombinant Factor VIII (rVWF:rFVIII) and rVWF in the Treatment of Bleeding Episodes in Subjects Diagnosed With Von Willebrand Disease
Query!
Secondary ID [1]
0
0
2010-024108-84
Query!
Secondary ID [2]
0
0
071001
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Von Willebrand Disease
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Blood
0
0
0
0
Query!
Clotting disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Other - Recombinant von Willebrand factor (rVWF)
Treatment: Drugs - Placebo
Treatment: Other - Recombinant factor VIIII (rFVIII)
Experimental: PK 80 Arm (minimum of 22 subjects with severe VWD) - PK assessment (80 IU/kg rVWF) + 12-month treatment period
Experimental: PK 50 Arm (14 subjects with type 3 VWD) - Two single-blinded PK assessments (50 IU/kg rVWF + rFVIII/placebo) + 12-month treatment period
Experimental: PK 50 Only Arm (minimum of 7 subjects with type 3 VWD) - PK assessment (50 IU/kg rVWF) only, no treatment of bleeding episodes
Experimental: Treatment Only (up to 7 subjects independent of VWD subtype) - Treatment of bleeding episodes for a total of 12 months
Treatment: Other: Recombinant von Willebrand factor (rVWF)
Intravenous administration
Treatment: Drugs: Placebo
Syringe supplied with physiologic saline solution for infusion
Treatment: Other: Recombinant factor VIIII (rFVIII)
Intravenous administration
Query!
Intervention code [1]
0
0
Treatment: Other
Query!
Intervention code [2]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Percentage of Participants With Treatment Success for Treated Bleeding Episodes
Query!
Assessment method [1]
0
0
Treatment success was defined as the extent of control of bleeding episodes (BEs) using a mean efficacy rating score of \<2.5 for a participant's BEs treated with study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) during the study period. Scores used: Excellent = 1 - actual infusions = estimated number of infusions required to treat BE; no additional VWF required (all BEs); Good = 2 - \>1-2 infusions (minor/moderate BEs) or \<1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs); Moderate = 3 = 3 infusions (minor/moderate BEs) or = 1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs); None = 4 - severe uncontrolled bleeding or intensity of bleeding not changed; additional VWF required. Included participants with available primary efficacy rating (prospective-excluding gastrointestinal bleeds) in the Full Analysis Set.
Query!
Timepoint [1]
0
0
For 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [1]
0
0
Percentage of Treated Bleeding Episodes With an Efficacy Rating of "Excellent" or "Good"
Query!
Assessment method [1]
0
0
Efficacy ratings "excellent" or "good" for the control of bleeding episodes (BEs) with study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) are defined as follows: Excellent - actual infusions = estimated number of infusions required to treat BE; no additional von Willebrand Factor (VWF) required (all BEs); Good - \>1-2 infusions (minor/moderate BEs) or \<1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs). The data set included prospectively estimated BEs treated with study product with an available efficacy rating from participants in the Full Analysis Set
Query!
Timepoint [1]
0
0
For 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [2]
0
0
Percentage of Treated Bleeding Episodes With an Efficacy Rating of "Excellent" or "Good", Excluding Gastrointestinal Bleeds
Query!
Assessment method [2]
0
0
Efficacy ratings of "excellent" or "good" for the control of bleeding episodes (BEs) with study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) are defined as follows: Excellent - actual infusions = estimated number of infusions required to treat BE; no additional von Willebrand Factor (VWF) required (all BEs); Good - \>1-2 infusions (minor/moderate BEs) or \<1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs). The data set included prospectively estimated BEs excluding gastrointestinal (GI) bleeds treated with study product with an available efficacy rating from participants in the Full Analysis Set.
Query!
Timepoint [2]
0
0
For 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [3]
0
0
Number of Infusions of rVWF:rFVIII and/or rVWF Per Bleeding Episode
Query!
Assessment method [3]
0
0
The actual number of infusions of recombinant von Willebrand factor:recombinant factor VIII (rVWF:rFVIII) and/or rVWF required to treat a bleeding episode (BE). BEs were to be initially treated with an infusion of rVWF:rFVIII and subsequently with rVWF with or without rFVIII, based on FVIII levels, if available. In cases, where no FVIII levels were available, the individual participant's PK data was used to determine the rFVIII dose. The data set included prospectively estimated BEs treated with study product with an available efficacy rating from participants in the Full Analysis Set.
Query!
Timepoint [3]
0
0
For 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [4]
0
0
Number of Units of rVWF:rFVIII and/or rVWF Per Bleeding Episode
Query!
Assessment method [4]
0
0
The number of units is provided as the actual dose \[IU/kg\] of recombinant von Willebrand factor:recombinant factor VIII (rVWF:rFVIII) and/or rVWF required to treat a bleeding episode (BE). BEs were to be initially treated with an infusion of rVWF:rFVIII and subsequently with rVWF with or without rFVIII, based on FVIII levels, if available. In cases, where no FVIII levels were available, the individual participant's PK data was used to determine the rFVIII dose. The data set included prospectively estimated BEs treated with study product of known lot number with an available efficacy rating from participants in the Full Analysis Set.
Query!
Timepoint [4]
0
0
For 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [5]
0
0
Percentage of Participants Who Develop Inhibitory Antibodies to FVIII
Query!
Assessment method [5]
0
0
Development of neutralizing antibodies (inhibitors) to factor VIII (FVIII) was assessed by the Nijmegen modification of the Bethesda assay. Positive FVIII inhibitor tests were defined as = 0.4 Bethesda units/mL (BU/mL) by the Nijmegen-modified Bethesda assay that is confirmed by a second test performed on an independent sample obtained 2-4 weeks following the first test. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [5]
0
0
For 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [6]
0
0
Percentage of Participants Who Develop Inhibitory Antibodies to VWF
Query!
Assessment method [6]
0
0
Neutralizing antibodies (inhibitors) to Von Willebrand Factor Ristocetin cofactor (VWF:RCo), VWF collagen binding (VWF:CB) and VWF Factor VIII binding (VWF:FVIIIB) activities were measured using Nijmegen modification of the Bethesda assay. One Bethesda Unit (BU) is thereby defined as the amount of inhibitor that decreased the measured activity in the assays to 50% of that of the negative control samples. The assays were validated using human plasma samples from two type 3 VWD patients with low (1-2 BU/mL) and high (\~10 BU/mL) titer inhibitors and plasma samples from non-human primates immunized with human rVWF (\>100 BU/mL). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [6]
0
0
After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [7]
0
0
Percentage of Participants Who Develop Binding Antibodies to VWF
Query!
Assessment method [7]
0
0
The presence of total binding anti-VWF antibodies was determined by an enzyme-linked immunosorbent assay (ELISA) employing polyclonal anti-human immunoglobulin (Ig) antibodies (IgG, IgM and IgA). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [7]
0
0
After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [8]
0
0
Percentage of Participants Who Develop Binding Antibodies to CHO
Query!
Assessment method [8]
0
0
The presence of total binding anti-CHO antibodies was determined by measuring total immunoglobulin (Ig) antibodies (IgG, IgA, IgM) against Chinese Hamster Ovary (CHO) protein using an enzyme-linked immunosorbent assay (ELISA). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [8]
0
0
After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [9]
0
0
Percentage of Participants Who Develop Binding Antibodies to rFurin
Query!
Assessment method [9]
0
0
The presence of total binding anti-rFurin antibodies was determined by measuring total immunoglobulin (Ig) antibodies (IgG, IgA, IgM) against rFurin protein using an enzyme-linked immunosorbent assay (ELISA). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [9]
0
0
After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [10]
0
0
Percentage of Participants Who Develop Binding Antibodies to Mouse Immunoglobulin
Query!
Assessment method [10]
0
0
The presence of total binding anti-Murine immunoglobulin (IgG) antibodies was determined using an enzyme-linked immunosorbent assay (ELISA). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [10]
0
0
After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [11]
0
0
Percentage of Participants Who Had an Occurrence of Thrombotic Events
Query!
Assessment method [11]
0
0
Query!
Timepoint [11]
0
0
After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [12]
0
0
Number of Adverse Events Related to Study Product Including Clinically Significant Changes in Laboratory Parameters and Vital Signs
Query!
Assessment method [12]
0
0
Adverse Events (AEs) related to study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) are described. Only laboratory parameters (hematology and clinical chemistry) and vital signs (physical examination, ECG) with clinically significant findings that are recorded as AEs are included. Categories presented as Severity-System Organ Class-Preferred Term Seriousness: serious adverse event (SAE); non serious adverse event (nsAE) System Organ Class: Cardiac disorders (CARD); General disorders and administration site conditions (GEN); Investigations (INV); Nervous system disorders (NERV); Skin and subcutaneous tissue disorders (SKN); Vascular disorders (VAS). Category title includes number of AEs \[N\] for the category.
Query!
Timepoint [12]
0
0
For 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [13]
0
0
Number of Participants With Adverse Events Related to Study Product Including Clinically Significant Changes in Laboratory Parameters and Vital Signs
Query!
Assessment method [13]
0
0
Number of participants with Adverse Events (AEs) related to study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) are described. Only laboratory parameters (hematology and clinical chemistry) and vital signs (physical examination, ECG) with clinically significant findings that are recorded as AEs are included. Categories presented as Severity-System Organ Class-Preferred Term Seriousness: serious adverse event (SAE); non serious adverse event (nsAE) System Organ Class: Cardiac disorders (CARD); General disorders and administration site conditions (GEN); Investigations (INV); Nervous system disorders (NERV); Skin and subcutaneous tissue disorders (SKN); Vascular disorders (VAS).
Query!
Timepoint [13]
0
0
For 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [14]
0
0
Number of Adverse Events by Infusion Related to Study Product Including Clinically Significant Changes in Laboratory Parameters and Vital Signs
Query!
Assessment method [14]
0
0
Adverse Events (AEs) by infusion related to study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) are described. Only laboratory parameters (hematology and clinical chemistry) and vital signs (physical examination, ECG) with clinically significant findings that are recorded as AEs are included. Categories presented as Severity-System Organ Class-Preferred Term Seriousness: serious adverse event (SAE); non serious adverse event (nsAE) System Organ Class: Cardiac disorders (CARD); General disorders and administration site conditions (GEN); Investigations (INV); Nervous system disorders (NERV); Skin and subcutaneous tissue disorders (SKN); Vascular disorders (VAS).
Query!
Timepoint [14]
0
0
For 12 months after first infusion of rVWF:rFVIII or rVWF
Query!
Secondary outcome [15]
0
0
PK50 - Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-8/Dose) of VWF:RCo
Query!
Assessment method [15]
0
0
Area under the plasma concentration curve (AUC) from time 0 to infinity of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for subjects in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [15]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [16]
0
0
PK50 - Area Under the Plasma Concentration/Time Curve From Time 0 to 96 Hours (AUC0-96h/Dose) of VWF:RCo
Query!
Assessment method [16]
0
0
Area under the plasma concentration curve (AUC) from time 0 to 96 hours of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [16]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [17]
0
0
PK50 - Mean Residence Time of VWF:RCo
Query!
Assessment method [17]
0
0
Mean Residence Time (MRT) of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [17]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [18]
0
0
PK50 - Clearance of VWF:RCo
Query!
Assessment method [18]
0
0
Clearance (CL) of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [18]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [19]
0
0
PK50 - Incremental Recovery of VWF:RCo
Query!
Assessment method [19]
0
0
Incremental Recovery (IR) at the maximum plasma concentration of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [19]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [20]
0
0
PK50 - Elimination Phase Half-Life of VWF:Co
Query!
Assessment method [20]
0
0
Elimination Phase Half-Life (T1/2) of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant FVIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [20]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [21]
0
0
PK50 - Volume of Distribution at Steady State of VWF:RCo
Query!
Assessment method [21]
0
0
Volume of Distribution at Steady State (Vss) of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [21]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [22]
0
0
PK50 - Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-8/Dose) of VWF:Ag
Query!
Assessment method [22]
0
0
Area under the plasma concentration curve (AUC) from time 0 to infinity of von Willebrand Factor Antigen (VWF:Ag) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [22]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [23]
0
0
PK50 - Area Under the Plasma Concentration/Time Curve From Time 0 to 96 Hours (AUC0-96h/Dose) of VWF:Ag
Query!
Assessment method [23]
0
0
Area under the plasma concentration curve (AUC) from time 0 to 96 hours of von Willebrand Factor Antigen (VWF:Ag) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [23]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout
Query!
Secondary outcome [24]
0
0
PK50 - Mean Residence Time of VWF:Ag
Query!
Assessment method [24]
0
0
Mean Residence Time (MRT) of von Willebrand Factor Antigen (VWF:Ag) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\] or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [24]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [25]
0
0
PK50 - Clearance of VWF:Ag
Query!
Assessment method [25]
0
0
Clearance (CL) of von Willebrand Factor Antigen (VWF:Ag) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [25]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [26]
0
0
PK50 - Incremental Recovery of VWF:Ag
Query!
Assessment method [26]
0
0
Incremental Recovery (IR) at the maximum plasma concentration of von Willebrand Factor Antigen (VWF:Ag) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [26]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [27]
0
0
PK50 - Elimination Phase Half-Life of VWF:Ag
Query!
Assessment method [27]
0
0
Elimination Phase Half-Life (T1/2) of von Willebrand Factor Antigen (VWF:Ag) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant FVIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category
Query!
Timepoint [27]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [28]
0
0
PK50 - Volume of Distribution at Steady State of VWF:Ag
Query!
Assessment method [28]
0
0
Volume of Distribution at Steady State (Vss) of von Willebrand Factor Antigen (VWF:Ag) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [28]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [29]
0
0
PK50 - Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-8/Dose) of VWF:CB
Query!
Assessment method [29]
0
0
Area under the plasma concentration curve (AUC) from time 0 to infinity of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [29]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [30]
0
0
PK50 - Area Under the Plasma Concentration/Time Curve From Time 0 to 96 Hours (AUC0-96h/Dose) of VWF:CB
Query!
Assessment method [30]
0
0
Area under the plasma concentration curve (AUC) from time 0 to 96 hours of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [30]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [31]
0
0
PK50 - Mean Residence Time of VWF:CB
Query!
Assessment method [31]
0
0
Mean Residence Time (MRT) of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [31]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [32]
0
0
PK50 - Clearance of VWF:CB
Query!
Assessment method [32]
0
0
Clearance (CL) of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [32]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [33]
0
0
PK50 - Incremental Recovery of VWF:CB
Query!
Assessment method [33]
0
0
Incremental Recovery (IR) at the maximum plasma concentration of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [33]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [34]
0
0
PK50 - Elimination Phase Half-Life of VWF:CB
Query!
Assessment method [34]
0
0
Elimination Phase Half-Life (T1/2) of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant FVIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [34]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [35]
0
0
PK50 - Volume of Distribution at Steady State of VWF:CB
Query!
Assessment method [35]
0
0
Volume of Distribution at Steady State (Vss) of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants\[N\] who provided data for the category.
Query!
Timepoint [35]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [36]
0
0
PK50 - Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-8/Dose) of FVIII:C
Query!
Assessment method [36]
0
0
Area under the plasma concentration curve (AUC) from time 0 to infinity of Factor VIII activity (FVIII:C) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [36]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [37]
0
0
PK50 - Area Under the Plasma Concentration/Time Curve From Time 0 to 96 Hours (AUC0-96h/Dose) of FVIII:C
Query!
Assessment method [37]
0
0
Area under the plasma concentration curve (AUC) from time 0 to 96 hours of Factor VIII activity (FVIII:C) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\], or 50 IU/kg VWF:RCo rVWF administered together with saline (placebo) \[rVWF\] for participants in the PK50 arms (Arm 1 and Arm 2). Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [37]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [38]
0
0
PK50 - Mean Residence Time of FVIII:C
Query!
Assessment method [38]
0
0
Mean Residence Time (MRT) of Factor VIII activity (FVIII:C) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\] for participants in the PK50 arms (Arm 1 and Arm 2).
Query!
Timepoint [38]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [39]
0
0
PK50 - Clearance of FVIII:C
Query!
Assessment method [39]
0
0
Clearance (CL) of Factor VIII activity (FVIII:C) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\] for participants in the PK50 arms (Arm 1 and Arm 2).
Query!
Timepoint [39]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [40]
0
0
PK50 - Incremental Recovery of FVIII:C
Query!
Assessment method [40]
0
0
Incremental Recovery (IR) at the maximum plasma concentration of Factor VIII activity (FVIII:C) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\] for participants in the PK50 arms (Arm 1 and Arm 2).
Query!
Timepoint [40]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [41]
0
0
PK50 - Elimination Phase Half-Life of FVIII:C
Query!
Assessment method [41]
0
0
Elimination Phase Half-Life (T1/2) of Factor VIII activity (FVIII:C) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant FVIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\] for participants in the PK50 arms (Arm 1 and Arm 2).
Query!
Timepoint [41]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [42]
0
0
PK50 - Volume of Distribution at Steady State of FVIII:C
Query!
Assessment method [42]
0
0
Volume of Distribution at Steady State (Vss) of Factor VIII activity (FVIII:C) after infusion of 50 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) administered together with 38.5 IU/kg recombinant Factor VIII (rFVIII) (ratio of 1.3:1±0.2) \[rVWF:rFVIII\] for participants in the PK50 arms (Arm 1 and Arm 2).
Query!
Timepoint [42]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [43]
0
0
PK80 - Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-8/Dose) of VWF:RCo
Query!
Assessment method [43]
0
0
Area under the plasma concentration curve (AUC) from time 0 to infinity of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [43]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [44]
0
0
PK80 - Area Under the Plasma Concentration/Time Curve From Time 0 to 96 Hours (AUC0-96h/Dose) of VWF:RCo
Query!
Assessment method [44]
0
0
Area under the plasma concentration curve (AUC) from time 0 to 96 hours of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [44]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [45]
0
0
PK80 - Mean Residence Time of VWF:RCo
Query!
Assessment method [45]
0
0
Mean Residence Time (MRT) of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [45]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [46]
0
0
PK80 - Clearance of VWF:RCo
Query!
Assessment method [46]
0
0
Clearance (CL) of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [46]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [47]
0
0
PK80 - Incremental Recovery of VWF:RCo
Query!
Assessment method [47]
0
0
Incremental Recovery (IR) at the maximum plasma concentration Area under the plasma concentration curve (AUC) from time 0 to infinity of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [47]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [48]
0
0
PK80 - Elimination Phase Half-Life of VWF:Co
Query!
Assessment method [48]
0
0
Elimination Phase Half-Life (T1/2) of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category
Query!
Timepoint [48]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [49]
0
0
PK80 - Volume of Distribution at Steady State of VWF:RCo
Query!
Assessment method [49]
0
0
Volume of Distribution at Steady State (Vss) of von Willebrand Factor Ristocetin cofactor (VWF:RCo) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study. PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [49]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [50]
0
0
PK80 - Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-8/Dose) of VWF:Ag
Query!
Assessment method [50]
0
0
Area under the plasma concentration curve (AUC) from time 0 to infinity of von Willebrand Factor Antigen (VWF:Ag) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [50]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [51]
0
0
PK80 - Area Under the Plasma Concentration/Time Curve From Time 0 to 96 Hours (AUC0-96h/Dose) of VWF:Ag
Query!
Assessment method [51]
0
0
Area under the plasma concentration curve (AUC) from time 0 to 96 hours of von Willebrand Factor Antigen (VWF:Ag) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [51]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [52]
0
0
PK80 - Mean Residence Time of VWF:Ag
Query!
Assessment method [52]
0
0
Mean Residence Time (MRT) of von Willebrand Factor Antigen (VWF:Ag) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [52]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [53]
0
0
PK80 - Clearance of VWF:Ag
Query!
Assessment method [53]
0
0
Clearance (CL) of von Willebrand Factor Antigen (VWF:Ag) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [53]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [54]
0
0
PK80 - Incremental Recovery of VWF:Ag
Query!
Assessment method [54]
0
0
Incremental Recovery (IR) at the maximum plasma concentration Area under the plasma concentration curve (AUC) from time 0 to infinity of von Willebrand Factor Antigen (VWF:Ag) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [54]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [55]
0
0
PK80 - Elimination Phase Half-Life of VWF:Ag
Query!
Assessment method [55]
0
0
Elimination Phase Half-Life (T1/2) of von Willebrand Factor Antigen (VWF:Ag) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [55]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [56]
0
0
PK80 - Volume of Distribution at Steady State of VWF:Ag
Query!
Assessment method [56]
0
0
Volume of Distribution at Steady State (Vss) of von Willebrand Factor Antigen (VWF:Ag) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [56]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [57]
0
0
PK80 - Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-8/Dose) of VWF:CB
Query!
Assessment method [57]
0
0
Area under the plasma concentration curve (AUC) from time 0 to infinity of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [57]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [58]
0
0
PK80 - Area Under the Plasma Concentration/Time Curve From Time 0 to 96 Hours (AUC0-96h/Dose) of VWF:CB
Query!
Assessment method [58]
0
0
Area under the plasma concentration curve (AUC) from time 0 to 96 hours of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participatns from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [58]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [59]
0
0
PK80 - Mean Residence Time of VWF:CB
Query!
Assessment method [59]
0
0
Mean Residence Time (MRT) of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [59]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [60]
0
0
PK80 - Clearance of VWF:CB
Query!
Assessment method [60]
0
0
Clearance (CL) of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [60]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [61]
0
0
PK80 - Incremental Recovery of VWF:CB
Query!
Assessment method [61]
0
0
Incremental Recovery (IR) at the maximum plasma concentration Area under the plasma concentration curve (AUC) from time 0 to infinity of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [61]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [62]
0
0
PK80 - Elimination Phase Half-Life of VWF:CB
Query!
Assessment method [62]
0
0
Elimination Phase Half-Life (T1/2) of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [62]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [63]
0
0
PK80 - Volume of Distribution at Steady State of VWF:CB
Query!
Assessment method [63]
0
0
Volume of Distribution at Steady State (Vss) of von Willebrand Factor Collagen Binding (VWF:CB) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [63]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [64]
0
0
PK80 - Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-8/Dose) of FVIII:C
Query!
Assessment method [64]
0
0
Area under the plasma concentration curve (AUC) from time 0 to infinity of Factor VIII activity (FVIII:C) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [64]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [65]
0
0
PK80 - Area Under the Plasma Concentration/Time Curve From Time 0 to 96 Hours (AUC0-96h/Dose) of FVIII:C
Query!
Assessment method [65]
0
0
Area under the plasma concentration curve (AUC) from time 0 to 96 hours of Factor VIII activity (FVIII:C) after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. Category title includes number of participants \[N\] who provided data for the category.
Query!
Timepoint [65]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Secondary outcome [66]
0
0
PK80- Ratio of Intra-participant PK of VWF:RCo, VWF:Ag and VWF:CB at Baseline and After 6 Months
Query!
Assessment method [66]
0
0
Area under the plasma concentration curve (AUC) from time 0 to infinity per dose (AUC0-8/dose) for von Willebrand Factor Ristocetin cofactor (VWF:RCo), von Willebrand Factor Antigen (VWF:Ag) and von Willebrand Factor Collagen Binding (VWF:CB). Each parameter was compared between the two PK assessments after infusion of 80 IU/kg recombinant von Willebrand Factor:von Willebrand Factor Ristocetin cofactor (VWF:RCo rVWF) \[rVWF\] for participants in the PK80 arm (participants from Arm 3 with PK80 data only). PK assessment conducted at first infusion of 80 IU/kg rWVF \[PK1\] and the second infusion of 80 IU/kg rVWF after participants were treated on demand for bleeding episodes for at least 6 months since their first infusion of study product \[PK2\]. 13 participants had data available for this endpoint i.e. data for PK1 and PK2.
Query!
Timepoint [66]
0
0
PK evaluations at pre-infusion, then at 15, 30 and 60 mins, and 3, 6, 12, 24, 30, 48, 72 and 96 hrs post-infusion. PK evaluation timeframe for 28 ± 3 days after first infusion of study product which includes PK evaluation for both infusions and washout.
Query!
Eligibility
Key inclusion criteria
* Participant has been diagnosed with:
1. Type 1 (Von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) < 20 IU/dL) or,
2. Type 2A (VWF:RCo < 20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (Factor VIII activity (FVIII:C) <10% and historically documented genetics), Type 2M or,
3. Type 3 ( Von Willebrand factor antigen (VWF:Ag) = 3 IU/dL) or,
4. Severe Von Willebrand disease (VWD) with a history of requiring substitution therapy with von Willebrand factor concentrate to control bleeding
* Participant, who participates in the treatment for bleeding episodes, has had a minimum of 1 documented bleed (medical history) requiring VWF coagulation factor replacement therapy during the previous 12 months prior to enrollment.
* Participant has a Karnofsky score = 60%
* Participant is at least 18 and not older than 65 years of age at enrollment
* If female of childbearing potential, participant presents with a negative pregnancy test
* Participant agrees to employ adequate birth control measures for the duration of the study
* Participant is willing and able to comply with the requirements of the protocol
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
65
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
* Participant has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (eg qualitative and quantitative platelet disorders or elevated PT/international normalized ratio [INR] >1.4).
* Participant has a documented history of a VWF:RCo half-life of <6 hours.
* Participant has a history or presence of a VWF inhibitor at screening.
* Participant has a history or presence of a factor VIII (FVIII) inhibitor with a titer =0.4 BU (by Nijmegen assay) or = 0.6 BU (by Bethesda assay).
* Participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.
* Participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.
* Participant has a medical history of a thromboembolic event.
* Participant is HIV positive with an absolute CD4 count <200/mm3.
* Participant has been diagnosed with cardiovascular disease (New York Heart Association [NYHA] classes 1-4.
* Participant has an acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, non-seasonal asthma) at screening.
* Participant has been diagnosed with significant liver disease as evidenced by any of the following: serum alanine aminotransferase (ALT) 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices).
* Participant has been diagnosed with renal disease, with a serum creatinine level =2 mg/dL.
* In the judgment of the investigator, the participant has another clinically significant concomitant disease (eg, uncontrolled hypertension) that may pose additional risks for the participant.
* Participant has been treated with an immunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to enrollment
* Participant is pregnant or lactating at the time of enrollment.
* Participant has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an investigational product or investigational device during the course of this study.
* Participant has a history of drug or alcohol abuse within the 2 years prior to enrollment.
* Participant has a progressive fatal disease and/or life expectancy of less than 3 months.
* Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures.
* Participant suffers from a mental condition rendering him/her unable to understand the nature, scope and possible consequences of the study and/or evidence of an uncooperative attitude.
* Participant is in prison or compulsory detention by regulatory and/or juridical order
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Non-randomised trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Crossover
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/11/2011
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/02/2014
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
49
Query!
Recruitment in Australia
Recruitment state(s)
SA,WA
Query!
Recruitment hospital [1]
0
0
Royal Adelaide Hospital - Adelaide
Query!
Recruitment hospital [2]
0
0
Royal Perth Hospital - Perth
Query!
Recruitment postcode(s) [1]
0
0
5000 - Adelaide
Query!
Recruitment postcode(s) [2]
0
0
6847 - Perth
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Florida
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Illinois
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Indiana
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
New Jersey
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
New York
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
North Carolina
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Pennsylvania
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Wisconsin
Query!
Country [10]
0
0
Austria
Query!
State/province [10]
0
0
Vienna
Query!
Country [11]
0
0
Belgium
Query!
State/province [11]
0
0
Bruxelles
Query!
Country [12]
0
0
Belgium
Query!
State/province [12]
0
0
Leuven
Query!
Country [13]
0
0
Bulgaria
Query!
State/province [13]
0
0
Sofia
Query!
Country [14]
0
0
Bulgaria
Query!
State/province [14]
0
0
Varna
Query!
Country [15]
0
0
Canada
Query!
State/province [15]
0
0
Alberta
Query!
Country [16]
0
0
Germany
Query!
State/province [16]
0
0
Berlin
Query!
Country [17]
0
0
Germany
Query!
State/province [17]
0
0
Duisburg
Query!
Country [18]
0
0
Germany
Query!
State/province [18]
0
0
Mainz
Query!
Country [19]
0
0
India
Query!
State/province [19]
0
0
Pune
Query!
Country [20]
0
0
India
Query!
State/province [20]
0
0
Vellore
Query!
Country [21]
0
0
Italy
Query!
State/province [21]
0
0
Florence
Query!
Country [22]
0
0
Italy
Query!
State/province [22]
0
0
Milano
Query!
Country [23]
0
0
Italy
Query!
State/province [23]
0
0
Padova
Query!
Country [24]
0
0
Italy
Query!
State/province [24]
0
0
Roma
Query!
Country [25]
0
0
Italy
Query!
State/province [25]
0
0
Vicenza
Query!
Country [26]
0
0
Japan
Query!
State/province [26]
0
0
Nagoya
Query!
Country [27]
0
0
Japan
Query!
State/province [27]
0
0
Tokyo
Query!
Country [28]
0
0
Netherlands
Query!
State/province [28]
0
0
Rotterdam
Query!
Country [29]
0
0
Netherlands
Query!
State/province [29]
0
0
Utrecht
Query!
Country [30]
0
0
Poland
Query!
State/province [30]
0
0
Gdansk
Query!
Country [31]
0
0
Poland
Query!
State/province [31]
0
0
Krakow
Query!
Country [32]
0
0
Poland
Query!
State/province [32]
0
0
Warsaw
Query!
Country [33]
0
0
Poland
Query!
State/province [33]
0
0
Wroclaw
Query!
Country [34]
0
0
Russian Federation
Query!
State/province [34]
0
0
Ekaterinburg
Query!
Country [35]
0
0
Russian Federation
Query!
State/province [35]
0
0
Kirov
Query!
Country [36]
0
0
Russian Federation
Query!
State/province [36]
0
0
Krasnoyarsk
Query!
Country [37]
0
0
Russian Federation
Query!
State/province [37]
0
0
St. Petersburg
Query!
Country [38]
0
0
Spain
Query!
State/province [38]
0
0
Galicia
Query!
Country [39]
0
0
Spain
Query!
State/province [39]
0
0
Madrid
Query!
Country [40]
0
0
Spain
Query!
State/province [40]
0
0
Palma de Mallorca
Query!
Country [41]
0
0
Spain
Query!
State/province [41]
0
0
Santander
Query!
Country [42]
0
0
Sweden
Query!
State/province [42]
0
0
Gothenburg
Query!
Country [43]
0
0
Sweden
Query!
State/province [43]
0
0
Malmö
Query!
Country [44]
0
0
United Kingdom
Query!
State/province [44]
0
0
London
Query!
Country [45]
0
0
United Kingdom
Query!
State/province [45]
0
0
Manchester
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Baxalta now part of Shire
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
The purpose of this Phase 3 study is to assess the pharmacokinetics of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary von Willebrand disease (VWD).
Query!
Trial website
https://clinicaltrials.gov/study/NCT01410227
Query!
Trial related presentations / publications
Gill JC, Castaman G, Windyga J, Kouides P, Ragni M, Leebeek FW, Obermann-Slupetzky O, Chapman M, Fritsch S, Pavlova BG, Presch I, Ewenstein B. Hemostatic efficacy, safety, and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease. Blood. 2015 Oct 22;126(17):2038-46. doi: 10.1182/blood-2015-02-629873. Epub 2015 Aug 3. Erratum In: Blood. 2016 Jun 2;127(22):2777. doi: 10.1182/blood-2016-04-711259. Blood. 2020 Nov 19;136(21):2479-2480. doi: 10.1182/blood.2020009144.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Study Director
Query!
Address
0
0
Takeda
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
Query!
What data in particular will be shared?
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
Query!
When will data be available (start and end dates)?
Query!
Available to whom?
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Query!
Available for what types of analyses?
Query!
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/takeda/
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
Type
Citations or Other Details
Journal
Gill JC, Castaman G, Windyga J, Kouides P, Ragni M...
[
More Details
]
Results are available at
https://clinicaltrials.gov/study/NCT01410227