Main
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Note: This record shows only 24 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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17 August 2021 |
Main ID: |
NCT02973087 |
Date of registration:
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22/11/2016 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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rVWF IN PROPHYLAXIS
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Scientific title:
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A PROSPECTIVE, PHASE 3, OPEN-LABEL, INTERNATIONAL MULTICENTER STUDY ON EFFICACY AND SAFETY OF PROPHYLAXIS WITH rVWF IN SEVERE VON WILLEBRAND DISEASE |
Date of first enrolment:
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November 16, 2017 |
Target sample size:
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29 |
Recruitment status: |
Completed |
URL:
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https://clinicaltrials.gov/show/NCT02973087 |
Study type:
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Interventional |
Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Prevention. Masking: None (Open Label).
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Phase:
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Phase 3
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Countries of recruitment
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Canada
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France
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Germany
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Italy
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Netherlands
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Russian Federation
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Spain
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Turkey
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United States
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Contacts
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Contact type:
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Name:
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Study Director |
Address:
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Telephone:
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Email:
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Affiliation:
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Takeda |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Participant has a documented diagnosis of severe von Willebrand disease (VWD)
(baseline Von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) less than (<)
20 International Units/Deciliter [IU/dL]) with a history of requiring substitution
therapy with von Willebrand factor concentrate to control bleeding
1. Type 1 (VWF:RCo <20 IU/dL) or,
2. Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype),
Type 2M or,
3. Type 3 (Von Willebrand factor antigen (VWF:Ag) less than or equal to [< or =] 3
IU/dL).
2. Diagnosis is confirmed by genetic testing and multimer analysis, documented in patient
history or at screening.
3. For on-demand patient group, participant currently receiving on-demand treatment for
whom prophylactic treatment is recommended by the investigator.
4. For Plasma derived von Willebrand factor (pdVWF) product switch patient group,
participant has been receiving prophylactic treatment of pdVWF products for no less
than 12 months prior to screening.
5. For on-demand patient group, participant has greater than or equal to (>or=) 3
documented spontaneous bleeds (not including menorrhagia) requiring von Willebrand
factor (VWF) treatment during the past 12 months.
6. Availability of records to reliably evaluate type, frequency and treatment of bleeding
episodes during at least 12 months preceding enrollment. Up to 24 months retrospective
data should be collected if available. Availability of dosing and factor consumption
during 12 months (up to 24 months) of treatment prior to enrollment is required for
pdVWF switch participants and is desired (but not a requirement) for on-demand
participants.
7. Participant is > or = 18 years old at the time of screening and has a body mass index
> or = 15 but <40 kilogram per meter square (kg/m^2).
8. If female of childbearing potential, participant presents with a negative blood/urine
pregnancy test at screening and agrees to employ adequate birth control measures for
the duration of the study.
9. Participant is willing and able to comply with the requirements of the protocol.
Exclusion Criteria:
1. The participant has been diagnosed with Type 2N Von Willebrand disease (VWD), pseudo
VWD, or another hereditary or acquired coagulation disorder other than VWD (eg
qualitative and quantitative platelet disorders or prothrombin time [PT]/international
normalized ratio [INR] greater than [>]1.4).
2. The participant is currently receiving prophylactic treatment with more than 5
infusions per week.
3. The participant is currently receiving prophylactic treatment with a weekly dose
exceeding 240 IU/kg.
4. The participant has a history or presence of a VWF inhibitor at screening.
5. The participant has a history or presence of a Factor VIII (FVIII) inhibitor with a
titer =0.4 Bethesda units (BU) (by Nijmegen modified Bethesda assay) or > or = 0.6
Bethesda Unit (BU) (by Bethesda assay).
6. The participant has a known hypersensitivity to any of the components of the study
drugs, such as to mouse or hamster proteins.
7. The participant has a medical history of immunological disorders, excluding seasonal
allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.
8. The participant has a medical history of a thromboembolic event.
9. The participant is human immunodeficiency virus (HIV) positive with an absolute Helper
T cell (CD4) count <200/ cubic millimeter (mm^3).
10. The participant has been diagnosed with significant liver disease per investigator's
medical assessment of the participant's current condition or medical history or as
evidenced by any of the following: serum alanine aminotransferase (ALT) greater than 5
times the upper limit of normal; hypoalbuminemia; portal vein hypertension (e.g.,
presence of otherwise unexplained splenomegaly, history of esophageal varices).
11. The participant has been diagnosed with renal disease, with a serum creatinine (CR)
level > or = 2.5 milligram per deciliter (mg/dL).
12. The participant has a platelet count <100,000/ milliliter (mL) at screening.
13. The participant has been treated with an immunomodulatory drug, excluding topical
treatment (e.g., ointments, nasal sprays), within 30 days prior to signing the
informed consent.
14. The participant is pregnant or lactating at the time of enrollment.
15. Patient has cervical or uterine conditions causing menorrhagia or metrorrhagia
(including infection, dysplasia).
16. The participant has participated in another clinical study involving another
Investigational product (IP) or investigational device within 30 days prior to
enrollment or is scheduled to participate in another clinical study involving an IP or
investigational device during the course of this study.
17. The participant has a progressive fatal disease and/or life expectancy of less than 15
months.
18. The participant is scheduled for a surgical intervention.
19. The participant is identified by the investigator as being unable or unwilling to
cooperate with study procedures.
20. The participant has 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.
21. The participant is in prison or compulsory detention by regulatory and/or juridical
order.
22. The participant is member of the study team or in a dependent relationship with one of
the study team members which includes close relatives (i.e., children, partner/spouse,
siblings and parents) as well as employees.
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Von Willebrand Disease
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Intervention(s)
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Biological: Antihemophilic Factor (Recombinant)
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Biological: von Willebrand factor (Recombinant)
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Primary Outcome(s)
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Ratio of Annualized Bleeding Rate (ABR) for Spontaneous Bleeding Episodes (BEs) (On-study ABR / Historical ABR) Assessed by Investigator During Prophylactic Treatment With rVWF Through Month 12
[Time Frame: Up to 12 months]
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Secondary Outcome(s)
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Pharmacodynamic (PD) Assessment: Maximum Plasma Concentration (Cmax) Based on Factor VIII Clotting (FVIII:C) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Clearance (CL) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Maximum Plasma Concentration (Cmax) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetics Assessment at Steady State: Maximum Plasma Concentration During the Partial Dosing Interval at Steady State (Cmax;ss) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Number of Participants Based on Severity of TEAEs
[Time Frame: From first dose of study drug up to end of study (approximately 32 months)]
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Percentage of Switch Participants With Spontaneous ABR Preservation Success Through Month 12
[Time Frame: Up to 12 months]
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Pharmacokinetic Assessment at Steady State: Area Under the Plasma Concentration Versus Time Curve From Time 0 to End of the Partial Dosing Interval (AUC0- Tau;ss) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Last Measurable Concentration (AUC0-tlast) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Mean Residence Time (MRT) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Volume of Distribution at Steady State (Vss) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetics Assessment at Steady State: Minimum Plasma Concentration During the Partial Dosing Interval at Steady State (Cmin;ss) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Total Number of Infusions Administered Per Participant During Prophylactic Treatment With rVWF Through Month 12
[Time Frame: Up to 12 months]
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Number of Participants Who Developed of Total Binding Antibodies to Von Willebrand Factor (rVWF) and Factor VIII (FVIII)
[Time Frame: Baseline through Month 12]
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Number of Participants With Clinically Significant Change From Baseline in Clinical Laboratory Parameters
[Time Frame: Baseline up to end of study (approximately 32 months)]
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Pharmacodynamic Assessment at Steady State: Minimum Plasma Concentration During the Partial Dosing Interval at Steady State (Cmin;ss) Based on Factor VIII Clotting (FVIII:C) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Incremental Recovery (IR) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Maximum Plasma Concentration (Cmax) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetics Assessment at Steady State: Minimum Time to Reach the Maximum Plasma Concentration at Steady State (Tmax;ss) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic (PK) Assessment: Incremental Recovery (IR) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Mean Residence Time (MRT) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Terminal Half-life (T1/2) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Number of Participants Who Developed Neutralizing Antibodies to Von Willebrand Factor (rVWF) and Factor VIII (FVIII)
[Time Frame: Baseline through Month 12]
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Pharmacokinetic Assessment: Volume of Distribution at Steady State (Vss) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Number of Treated Spontaneous BEs by Location of Bleeding While on Prophylactic Treatment With rVWF Through Month 12
[Time Frame: Up to 12 months]
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Pharmacokinetics Assessment at Steady State: Maximum Plasma Concentration During the Partial Dosing Interval at Steady State (Cmax;ss) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetics Assessment at Steady State: Minimum Time to Reach the Maximum Plasma Concentration at Steady State (Tmax;ss) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacodynamic Assessment at Steady State: Maximum Plasma Concentration During the Partial Dosing Interval at Steady State (Cmax;ss) Based on Factor VIII Clotting (FVIII:C) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity (AUC0-8) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Minimum Time to Reach the Maximum Plasma Concentration (Tmax) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Terminal Half-life (T1/2) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Total Weight Adjusted Consumption of rVWF Per Participant During Prophylactic Treatment Through Month 12
[Time Frame: Up to 12 months]
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Number of Participants With Clinically Significant Change From Baseline in Vital Signs
[Time Frame: Baseline up to end of study (approximately 32 months)]
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Number of Participants With TEAEs Based Causality
[Time Frame: From first dose of study drug up to end of study (approximately 32 months)]
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Percentage of Prior On-demand Participants Achieved Spontaneous ABR Percent Reduction Success Through Month 12
[Time Frame: Up to 12 months]
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Pharmacokinetic Assessment: Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity (AUC0-8) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Last Measurable Concentration (AUC0-tlast) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetics Assessment at Steady State: Minimum Plasma Concentration During the Partial Dosing Interval at Steady State (Cmin;ss) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetics Assessment at Steady State: Minimum Time to Reach the Maximum Plasma Concentration at Steady State (Tmax;ss) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Number of Participants Who Developed Binding Antibodies to Chinese Hamster Ovary (CHO) Proteins, Mouse Immunoglobulin G (IgG) and/or rFurin
[Time Frame: Baseline through Month 12]
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Number of Participants With Hypersensitivity Reactions
[Time Frame: From first dose of study drug up to end of study (approximately 32 months)]
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Number of Participants With Thromboembolic Events
[Time Frame: From first dose of study drug up to end of study (approximately 32 months)]
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Pharmacodynamic Assessment at Steady State: Area Under the Plasma Concentration Versus Time Curve From Time 0 to End of the Partial Dosing Interval (AUC0- Tau;ss) Based on Factor VIII Clotting (FVIII:C) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacodynamic Assessment at Steady State: Minimum Time to Reach the Maximum Plasma Concentration at Steady State (Tmax;ss) Based on Factor VIII Clotting (FVIII:C) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacodynamic Assessment: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Last Measurable Concentration (AUC0-tlast) Based on Factor VIII Clotting (FVIII:C) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacodynamic Assessment: Minimum Time to Reach the Maximum Plasma Concentration (Tmax) Based on Factor VIII Clotting (FVIII:C) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment at Steady State: Area Under the Plasma Concentration Versus Time Curve From Time 0 to End of the Partial Dosing Interval (AUC0- Tau;ss) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity (AUC0-8) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Clearance (CL) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Clearance (CL) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Incremental Recovery (IR) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Mean Residence Time (MRT) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Minimum Time to Reach the Maximum Plasma Concentration (Tmax) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Terminal Half-life (T1/2) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Volume of Distribution at Steady State (Vss) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Average Number of Infusions Per Week Per Participant During Prophylactic Treatment With rVWF Through Month 12
[Time Frame: Up to 12 months]
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Factor VIII (FVIII) Clotting Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Number of Participants Based on Categorized Spontaneous ABR (sABR) Through Month 12
[Time Frame: Baseline through Month 12]
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Number of Participants Reporting One or More Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
[Time Frame: From first dose of study drug up to end of study (approximately 32 months)]
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Pharmacokinetics Assessment at Steady State: Minimum Plasma Concentration During the Partial Dosing Interval at Steady State (Cmin;ss) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment at Steady State: Area Under the Plasma Concentration Versus Time Curve From Time 0 to End of the Partial Dosing Interval (AUC0- Tau;ss) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Last Measurable Concentration (AUC0-tlast) Based on Von Willebrand Factor Antigen (VWF:Ag) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Maximum Plasma Concentration (Cmax) Based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetic Assessment: Minimum Time to Reach the Maximum Plasma Concentration (Tmax) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At baseline: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Pharmacokinetics Assessment at Steady State: Maximum Plasma Concentration During the Partial Dosing Interval at Steady State (Cmax;ss) Based on Von Willebrand Factor Collagen Binding (VWF:CB) Activity
[Time Frame: At Month 12: Within 30 minutes pre-infusion; and post-infusion at 15, 30 minutes and 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours]
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Secondary ID(s)
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2016-001478-14
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071301
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available: |
Yes |
URL: |
https://clinicaltrials.gov/ct2/show/results/NCT02973087
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URL of the protocol: |
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Date Posted: |
06/08/2021 |
Date of completion: |
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Date of first publication: |
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Results summary: |
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Baseline characteristics: |
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Adverse events: |
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Outcome measures: |
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IPD sharing plan: |
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IPD sharing description: |
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