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Recombinant Von Willebrand Factor in Subjects With Severe Von Willebrand Disease Undergoing Surgery

Primary Purpose

Von Willebrand Disease

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Recombinant von Willebrand Factor (rVWF)

About this trial

This is an interventional treatment trial for Von Willebrand Disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Diagnosis of severe von Willebrand disease (VWD) as listed below and elective surgical procedure planned
1. Type 1 (Von Willebrand factor : Ristocetin cofactor activity (VWF:RCo) <20 IU/dL), or
2. Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype), Type 2N (FVIII:C<10% and historically documented genetics), Type 2M, or
3. Type 3 (Von Willebrand factor antigen (VWF:Ag) ≤ 3 IU/dL)
VWD with a history of requiring substitution therapy with von Willebrand factor (VWF) concentrate to control bleeding
If type 3 VWD (VWF Antigen /VWF:Ag ≤ 3 IU/dL), participant has a medical history of at least 20 exposure days to VWF/FVIII coagulation factor concentrates (including cryoprecipitate or fresh frozen plasma)
If type 1 or type 2 VWD, participant has a medical history of 5 exposure days or a past major surgery requiring VWF/FVIII coagulation factor concentrates (including cryoprecipitate or fresh frozen plasma)
Participant is at least 18 years of age
If female of childbearing potential, participant presents with a negative pregnancy test
If applicable, 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

Exclusion Criteria:

Diagnosis of pseudo VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time [PT] / international normalized ratio [INR] > 1.4)
History or presence of a VWF inhibitor at screening
History or presence of a factor VIII (FVIII) inhibitor with a titer ≥ 0.4 BU (Nijmegen-modified Bethesda assay ) or ≥ 0.6 BU (by Bethesda assay)
Known hypersensitivity to any of the components of the study drugs, such as to mouse or hamster proteins
Medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies
Medical history of a thromboembolic event
HIV positive with an absolute CD4 count < 200/mm3
Platelet count < 100,000/mL
Diagnosis of significant liver disease, as evidenced by, but not limited to, 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) or liver cirrhosis classified as Child B or C
Diagnosis of renal disease, with a serum creatinine level ≥ 2 .5mg/dL
Participant has been treated with an immunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to signing the informed consent
Participant is pregnant or lactating at the time informed content is obtained
Participant has participated in another clinical study involving an investigational product (IP), other than rVWF with or without ADVATE, 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. However, eligible patients participating in the rVWF Prophylaxis Study (071301) may be enrolled.
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
Participant is a member of the study team conducting this study or in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees.

Sites / Locations

University of Colorado Hemophilia & Thrombosis Center
University of Miami, Jackson Memorial Hospital
Georgia Regents University
John Hopkins University
Dana Farber Cancer Institute
Rutgers - Robert Wood Johnson Medical School
Duke University Medical Center
Case Western Reserve University Hospital
Cleveland Clinic
University of Oklahoma Health Sciences Center
Medical University of South Carolina (MUSC)
Blood Center of South East Wisconsin
Royal Adelaide Hospital
The Perth Blood Institute
Fiona Stanley Hospital
AKH - Medizinische Universität Wien
Fakultni nemocnice Ostrava
Universitätsmedizin der Johannes Gutenberg Universität Mainz
Azienda Ospedaliero - Universitaria Careggi
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Umberto I Policlinico di Roma, Universitá di Roma La Sapienza
Erasmus Medisch Centrum
Regional Budgetary State Healthcare Institution (SHI) "Regional Clinical Hospital"
FSI Kirov Institute of Hematology and Blood Transfusion FMBA
Complejo Hospitalario Universitario A Coruña
Hospital Universitari i Politècnic La Fe
Taichung Veterans General Hospital
Tri-Service General Hospital
National Taiwan University Hospital
Ege University Medical Faculty
SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
Derriford Hospital
Royal Free Hospital
Churchill Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Recombinant von Willebrand Factor (rVWF)

Arm Description

Surgery participants treated with Recombinant von Willebrand Factor (rVWF)

Outcomes

Primary Outcome Measures

Overall Hemostatic Efficacy as Assessed by the Investigator (Hemophilia Physician)

Hemostatic efficacy will be rated on a scale of excellent - good - moderate - none. Excellent: Intra-, and postoperative hemostasis achieved with rVWF with our without ADVATE was as good or better than that expected for the type of surgical procedure performed in a hemostatically normal subject. Good: Intra-, and postoperative hemostasis achieved with rVWF with or without ADVATE was probably as good as that expected for the type of surgical procedure performed in a hemostatically normal subject. Moderate: Intra-, and postoperative hemostasis with rVWF with or without ADVATE was clearly less than optimal for the type of procedure performed but was maintained without the need to change the rVWF concentrate. None: Participant experienced uncontrolled bleeding that was the result of inadequate therapeutic response despite proper dosing, necessitating a change of rVWF concentrate.

Secondary Outcome Measures

Intraoperative Actual Versus Predicted Blood Loss as Assessed by the Operating Surgeon

The predicted blood loss will be estimated preoperatively by the operating surgeon based on a hemostatically normal individual of the same sex, age, stature and co-morbidities as the participant. The actual blood loss will be assessed consisting of the estimated blood loss, including into swabs, towels and suction during the procedure, per the anesthesiologist's record.

Intraoperative Actual Blood Loss Relative to Predicted Blood Loss

Actual blood loss relative to predicted blood loss will be calculated as [Actual Blood loss (mL)] divided by [Predicted Blood Loss (mL) multiplied by 100.

Intraoperative Actual Versus Predicted Blood Loss Score as Assessed by the Operating Surgeon

Hemostatic efficacy will be rated on a scale of excellent - good - moderate - none. Excellent: Intraoperative blood loss was less than or equal to the maximum blood loss expected for the type of procedure performed in a hemostatically normal subject (≤ 100%). Good: Intraoperative blood loss was up to 50% more than the maximum expected blood loss for the type of procedure performed in a hemostatically normal subject (101-150%) Moderate: Intraoperative blood loss was more than 50% of the maximum expected blood loss for the type of procedure performed in a hemostatically normal subject (>150%). None: Uncontrolled hemorrhage that was the result of inadequate therapeutic response despite proper dosing, necessitating a change of clotting factor replacement regimen.

Intraoperative Hemostatic Efficacy Score as Assessed by the Operating Surgeon

Hemostatic efficacy will be rated on a scale of excellent - good - moderate - none. Excellent: Intraoperative hemostasis achieved with rVWF with our without ADVATE was as good or better than that expected for the type of surgical procedure performed in a hemostatically normal subject. Good: Intraoperative hemostasis achieved with rVWF with or without ADVATE was probably as good as that expected for the type of surgical procedure performed in a hemostatically normal subject. Moderate: Intraoperative hemostasis with rVWF with or without ADVATE was clearly less than optimal for the type of procedure performed but was maintained without the need to change the rVWF concentrate. None: Participant experienced uncontrolled bleeding that was the result of inadequate therapeutic response despite proper dosing, necessitating a change of rVWF concentrate.

Daily Intra- and Postoperative Weight-adjusted Dose of rVWF With or Without ADVATE

Occurrence of Adverse Events

Treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs) will be evaluated.

Occurrence of Thrombotic Events

Treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs) will be evaluated for thrombotic events.

Occurrence of Severe Allergic Reactions (eg, Anaphylaxis)

Treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs) will be evaluated for severe allergic reactions.

Number of Participants Who Developed Inhibitory and Total Binding Antibodies to Von Willebrand Factor (VWF) and Inhibitory Antibodies to Factor VIII (FVIII)

Participants were treated with recombinant van Willebrand Factor (rVWF) with or without ADVATE.

Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins, Mouse Immunoglobulin G (IgG) or Recombinant Furin (rFurin)

Participants were treated with recombinant van Willebrand Factor (rVWF) with or without ADVATE.

Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion (AUC 0-72 h/Dose)

This assessment is only required for subjects undergoing major surgery. Subjects will receive a PK infusion at a dose of 50±5 IU/kg rVWF:RCo within 42 days prior to surgery. The area under the plasma concentration/time curve from 0 to 72 hours post-infusion will be computed using the linear trapezoidal rule. For the calculation of AUC(0-72h) the levels at 72 hours will be linearly interpolated/extrapolated from the 2 nearest sampling time points. PK analysis was performed for the following analytes: VWF Ristocetin Cofactor Activity (VWF:RCo), VWF Antigen Activity (VWF:Ag), VWF Collagen Binding Activity (VWF:CB), VWF Activity Measured INNOVANCE VWF Ac Assay (VWF:Ac), FVIII Coagulation Activity (FVIII:C)

Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinity (AUC 0-∞ /Dose)

This assessment is only required for subjects undergoing major surgery. Subjects will receive a PK infusion at a dose of 50±5 IU/kg rVWF:RCo within 42 days prior to surgery. The area under the plasma concentration/time curve from time 0 to infinity and the area under the first moment curve from time 0 to infinity will be calculated as the sum of AUC or AUMC from time 0 to the time of last quantifiable concentration plus a tail area correction calculated as Ct/λz and Ct/λz(t+1/λz), respectively, where Ct is the last quantifiable concentration, t is the time of last quantifiable concentration and λz is the terminal or disposition rate constant. PK analysis was performed for the following analytes: VWF Ristocetin Cofactor Activity (VWF:RCo), VWF Antigen Activity (VWF:Ag), VWF Collagen Binding Activity (VWF:CB), VWF Activity Measured INNOVANCE VWF Ac Assay (VWF:Ac), FVIII Coagulation Activity (FVIII:C)

Pharmacokinetics: Mean Residence Time (MRT)

This assessment is only required for subjects undergoing major surgery. Subjects will receive a PK infusion at a dose of 50±5 IU/kg rVWF:RCo within 42 days prior to surgery. Mean residence time will be calculated as area under the first moment curve from time 0 to infinity divided by the area under the curve time 0 to infinity minus T/2 where T is the duration of the infusion. PK analysis was performed for the following analytes: VWF Ristocetin Cofactor Activity (VWF:RCo), VWF Antigen Activity (VWF:Ag), VWF Collagen Binding Activity (VWF:CB), VWF Activity Measured INNOVANCE VWF Ac Assay (VWF:Ac)

Pharmacokinetics: Clearance (CL)

This assessment is only required for subjects undergoing major surgery. Subjects will receive a PK infusion at a dose of 50±5 IU/kg rVWF:RCo within 42 days prior to surgery. Clearance will be calculated as dose (IU/kg) divided by the area under the curve time 0 to infinity. PK analysis was performed for the following analytes: VWF Ristocetin Cofactor Activity (VWF:RCo), VWF Antigen Activity (VWF:Ag), VWF Collagen Binding Activity (VWF:CB), VWF Activity Measured INNOVANCE VWF Ac Assay (VWF:Ac)

Pharmacokinetics: Incremental Recovery (IR)

This assessment is only required for subjects undergoing major surgery. Subjects will receive a PK infusion at a dose of 50±5 IU/kg rVWF:RCo within 42 days prior to surgery. Incremental recovery will be calculated as (Cmax minus Cpreinfusion) divided by the dose (IU/kg) where kg refers to the body weight at the time of dosing and Cmax is the observed maximum concentration before correction for pre-infusion values. PK analysis was performed for the following analytes: VWF Ristocetin Cofactor Activity (VWF:RCo), VWF Antigen Activity (VWF:Ag), VWF Collagen Binding Activity (VWF:CB), VWF Activity Measured INNOVANCE VWF Ac Assay (VWF:Ac)

Pharmacokinetics: Elimination Phase Half-life (T1/2)

This assessment is only required for subjects undergoing major surgery. Subjects will receive a PK infusion at a dose of 50±5 IU/kg rVWF:RCo within 42 days prior to surgery. Terminal or disposition half-life (T1/2) will be calculated as ln2/λz where λz is the terminal elimination rate constant as calculated in WinNonlin NCA using at least three quantifiable concentrations. PK analysis was performed for the following analytes: VWF Ristocetin Cofactor Activity (VWF:RCo), VWF Antigen Activity (VWF:Ag), VWF Collagen Binding Activity (VWF:CB), VWF Activity Measured INNOVANCE VWF Ac Assay (VWF:Ac)

Pharmacokinetics: Volume of Distribution at Steady State (Vss)

This assessment is only required for subjects undergoing major surgery. Subjects will receive a PK infusion at a dose of 50±5 IU/kg rVWF:RCo within 42 days prior to surgery. Vss will be calculated as the clearance multiplied with the mean residence time. PK analysis was performed for the following analytes: VWF Ristocetin Cofactor Activity (VWF:RCo), VWF Antigen Activity (VWF:Ag), VWF Collagen Binding Activity (VWF:CB), VWF Activity Measured INNOVANCE VWF Ac Assay (VWF:Ac)

Full Information

NCT ID

NCT02283268

First Posted

October 27, 2014

Last Updated

April 29, 2021

Sponsor

Baxalta now part of Shire

1. Study Identification

Unique Protocol Identification Number

NCT02283268

Brief Title

Recombinant Von Willebrand Factor in Subjects With Severe Von Willebrand Disease Undergoing Surgery

Official Title

A Phase 3, Prospective, Multicenter Study to Evaluate Efficacy and Safety of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Elective Surgical Procedures in Subjects With Severe Von Willebrand Disease

Study Type

Interventional

2. Study Status

Record Verification Date

April 2021

Overall Recruitment Status

Completed

Study Start Date

April 1, 2015 (Actual)

Primary Completion Date

July 6, 2016 (Actual)

Study Completion Date

July 6, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Baxalta now part of Shire

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The purpose of the study is to assess the efficacy and safety of recombinant von Willebrand factor (rVWF) with or without ADVATE in major and minor elective surgical procedures in adult patients with hereditary severe von Willebrand disease (VWD).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Von Willebrand Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

24 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Recombinant von Willebrand Factor (rVWF)

Arm Type

Experimental

Arm Description

Surgery participants treated with Recombinant von Willebrand Factor (rVWF)

Intervention Type

Biological

Intervention Name(s)

Recombinant von Willebrand Factor (rVWF)

Intervention Description

rVWF will be administered by intravenous bolus infusion. Participants planned for major surgery will undergo a baseline pharmacokinetic assessment prior to surgery. The peri- and postoperative substitution regimen will be individualized according to the PK results, intensity and duration of the hemostatic challenge, and the institution´s standard of care.

Primary Outcome Measure Information:

Title

Overall Hemostatic Efficacy as Assessed by the Investigator (Hemophilia Physician)

Description

Time Frame

24 hours after last peri-operative infusion or at completion of Day 14 (± 2 days) visit, whichever occurs earlier

Secondary Outcome Measure Information:

Title

Intraoperative Actual Versus Predicted Blood Loss as Assessed by the Operating Surgeon

Description

Time Frame

Day 0 (at completion of surgery)

Title

Intraoperative Actual Blood Loss Relative to Predicted Blood Loss

Description

Actual blood loss relative to predicted blood loss will be calculated as [Actual Blood loss (mL)] divided by [Predicted Blood Loss (mL) multiplied by 100.

Time Frame

Day 0 (at completion of surgery)

Title

Intraoperative Actual Versus Predicted Blood Loss Score as Assessed by the Operating Surgeon

Description

Time Frame

Day 0 (at completion of surgery)

Title

Intraoperative Hemostatic Efficacy Score as Assessed by the Operating Surgeon

Description

Time Frame

Day 0 (at completion of surgery)

Title

Daily Intra- and Postoperative Weight-adjusted Dose of rVWF With or Without ADVATE

Time Frame

Daily, from day of surgery through postoperative Day 14 (± 2 days)

Title

Occurrence of Adverse Events

Description

Treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs) will be evaluated.

Time Frame

From first infusion of investigational product through study completion (ie, 14 (± 2) days post surgery)

Title

Occurrence of Thrombotic Events

Description

Treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs) will be evaluated for thrombotic events.

Time Frame

From first infusion of investigational product through study completion (ie, 14 (± 2) days post surgery)

Title

Occurrence of Severe Allergic Reactions (eg, Anaphylaxis)

Description

Treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs) will be evaluated for severe allergic reactions.

Time Frame

From first infusion of investigational product through study completion (ie, 14 (± 2) days post surgery)

Title

Number of Participants Who Developed Inhibitory and Total Binding Antibodies to Von Willebrand Factor (VWF) and Inhibitory Antibodies to Factor VIII (FVIII)

Description

Participants were treated with recombinant van Willebrand Factor (rVWF) with or without ADVATE.

Time Frame

Testing occurred throughout the study at screening, prior PK infusion, pre-surgery, post surgery in case of excessive bleeding or unexplained bleeding, at postoperative day 7 and at study completion visit (ie. 14 (± 2) days post surgery).

Title

Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins, Mouse Immunoglobulin G (IgG) or Recombinant Furin (rFurin)

Description

Participants were treated with recombinant van Willebrand Factor (rVWF) with or without ADVATE.

Time Frame

Title

Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion (AUC 0-72 h/Dose)

Description

Time Frame

PK measurements were done within 30 minutes pre-infusion, and post infusion at 30 (± 5) minutes, 60 (± 5) minutes, 6 (± 1) hours, 12 (± 1) hours, 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours.

Title

Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinity (AUC 0-∞ /Dose)

Description

This assessment is only required for subjects undergoing major surgery. Subjects will receive a PK infusion at a dose of 50±5 IU/kg rVWF:RCo within 42 days prior to surgery. The area under the plasma concentration/time curve from time 0 to infinity and the area under the first moment curve from time 0 to infinity will be calculated as the sum of AUC or AUMC from time 0 to the time of last quantifiable concentration plus a tail area correction calculated as Ct/λz and Ct/λz(t+1/λz), respectively, where Ct is the last quantifiable concentration, t is the time of last quantifiable concentration and λz is the terminal or disposition rate constant. PK analysis was performed for the following analytes: VWF Ristocetin Cofactor Activity (VWF:RCo), VWF Antigen Activity (VWF:Ag), VWF Collagen Binding Activity (VWF:CB), VWF Activity Measured INNOVANCE VWF Ac Assay (VWF:Ac), FVIII Coagulation Activity (FVIII:C)

Time Frame

Title

Pharmacokinetics: Mean Residence Time (MRT)

Description

Time Frame

Title

Pharmacokinetics: Clearance (CL)

Description

Time Frame

Title

Pharmacokinetics: Incremental Recovery (IR)

Description

Time Frame

Title

Pharmacokinetics: Elimination Phase Half-life (T1/2)

Description

Time Frame

Title

Pharmacokinetics: Volume of Distribution at Steady State (Vss)

Description

Time Frame

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Diagnosis of severe von Willebrand disease (VWD) as listed below and elective surgical procedure planned Type 1 (Von Willebrand factor : Ristocetin cofactor activity (VWF:RCo) <20 IU/dL), or Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype), Type 2N (FVIII:C<10% and historically documented genetics), Type 2M, or Type 3 (Von Willebrand factor antigen (VWF:Ag) ≤ 3 IU/dL) VWD with a history of requiring substitution therapy with von Willebrand factor (VWF) concentrate to control bleeding If type 3 VWD (VWF Antigen /VWF:Ag ≤ 3 IU/dL), participant has a medical history of at least 20 exposure days to VWF/FVIII coagulation factor concentrates (including cryoprecipitate or fresh frozen plasma) If type 1 or type 2 VWD, participant has a medical history of 5 exposure days or a past major surgery requiring VWF/FVIII coagulation factor concentrates (including cryoprecipitate or fresh frozen plasma) Participant is at least 18 years of age If female of childbearing potential, participant presents with a negative pregnancy test If applicable, 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 Exclusion Criteria: Diagnosis of pseudo VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time [PT] / international normalized ratio [INR] > 1.4) History or presence of a VWF inhibitor at screening History or presence of a factor VIII (FVIII) inhibitor with a titer ≥ 0.4 BU (Nijmegen-modified Bethesda assay ) or ≥ 0.6 BU (by Bethesda assay) Known hypersensitivity to any of the components of the study drugs, such as to mouse or hamster proteins Medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies Medical history of a thromboembolic event HIV positive with an absolute CD4 count < 200/mm3 Platelet count < 100,000/mL Diagnosis of significant liver disease, as evidenced by, but not limited to, 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) or liver cirrhosis classified as Child B or C Diagnosis of renal disease, with a serum creatinine level ≥ 2 .5mg/dL Participant has been treated with an immunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to signing the informed consent Participant is pregnant or lactating at the time informed content is obtained Participant has participated in another clinical study involving an investigational product (IP), other than rVWF with or without ADVATE, 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. However, eligible patients participating in the rVWF Prophylaxis Study (071301) may be enrolled. 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 Participant is a member of the study team conducting this study or in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Study Director

Organizational Affiliation

Takeda

Official's Role

Study Director

Facility Information:

Facility Name

University of Colorado Hemophilia & Thrombosis Center

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

University of Miami, Jackson Memorial Hospital

City

Miami

State/Province

Florida

ZIP/Postal Code

33136

Country

United States

Facility Name

Georgia Regents University

City

Augusta

State/Province

Georgia

ZIP/Postal Code

30912

Country

United States

Facility Name

John Hopkins University

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21205

Country

United States

Facility Name

Dana Farber Cancer Institute

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02215

Country

United States

Facility Name

Rutgers - Robert Wood Johnson Medical School

City

New Brunswick

State/Province

New Jersey

ZIP/Postal Code

08901

Country

United States

Facility Name

Duke University Medical Center

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27705

Country

United States

Facility Name

Case Western Reserve University Hospital

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44106

Country

United States

Facility Name

Cleveland Clinic

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44195

Country

United States

Facility Name

University of Oklahoma Health Sciences Center

City

Oklahoma City

State/Province

Oklahoma

ZIP/Postal Code

73104

Country

United States

Facility Name

Medical University of South Carolina (MUSC)

City

Charleston

State/Province

South Carolina

ZIP/Postal Code

29425

Country

United States

Facility Name

Blood Center of South East Wisconsin

City

Milwaukee

State/Province

Wisconsin

ZIP/Postal Code

53233

Country

United States

Facility Name

Royal Adelaide Hospital

City

Adelaide

State/Province

South Australia

ZIP/Postal Code

5000

Country

Australia

Facility Name

The Perth Blood Institute

City

Nedlands

State/Province

Western Australia

ZIP/Postal Code

6009

Country

Australia

Facility Name

Fiona Stanley Hospital

City

Perth

State/Province

Western Australia

ZIP/Postal Code

6000

Country

Australia

Facility Name

AKH - Medizinische Universität Wien

City

Vienna

ZIP/Postal Code

1090

Country

Austria

Facility Name

Fakultni nemocnice Ostrava

City

Ostrava

ZIP/Postal Code

70852

Country

Czechia

Facility Name

Universitätsmedizin der Johannes Gutenberg Universität Mainz

City

Mainz

State/Province

Rheinland-Pfalz

ZIP/Postal Code

55101

Country

Germany

Facility Name

Azienda Ospedaliero - Universitaria Careggi

City

Firenze (Florence)

ZIP/Postal Code

50139

Country

Italy

Facility Name

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

City

Milano

ZIP/Postal Code

20122

Country

Italy

Facility Name

Umberto I Policlinico di Roma, Universitá di Roma La Sapienza

City

Rome

ZIP/Postal Code

00144

Country

Italy

Facility Name

Erasmus Medisch Centrum

City

Rotterdam

ZIP/Postal Code

3015 CE

Country

Netherlands

Facility Name

Regional Budgetary State Healthcare Institution (SHI) "Regional Clinical Hospital"

City

Barnaul

ZIP/Postal Code

656024

Country

Russian Federation

Facility Name

FSI Kirov Institute of Hematology and Blood Transfusion FMBA

City

Kirov

ZIP/Postal Code

610027

Country

Russian Federation

Facility Name

Complejo Hospitalario Universitario A Coruña

City

A Coruña

ZIP/Postal Code

15006

Country

Spain

Facility Name

Hospital Universitari i Politècnic La Fe

City

Valencia

ZIP/Postal Code

46026

Country

Spain

Facility Name

Taichung Veterans General Hospital

City

Taichung

ZIP/Postal Code

40705

Country

Taiwan

Facility Name

Tri-Service General Hospital

City

Taipei

ZIP/Postal Code

10002

Country

Taiwan

Facility Name

National Taiwan University Hospital

City

Taipei

ZIP/Postal Code

11490

Country

Taiwan

Facility Name

Ege University Medical Faculty

City

Izmir

ZIP/Postal Code

35100

Country

Turkey

Facility Name

SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

City

Lviv

ZIP/Postal Code

79044

Country

Ukraine

Facility Name

Derriford Hospital

City

Plymouth

State/Province

Devon

ZIP/Postal Code

PL6 8DH

Country

United Kingdom

Facility Name

Royal Free Hospital

City

London

State/Province

Greater London

ZIP/Postal Code

NW3 2QG

Country

United Kingdom

Facility Name

Churchill Hospital

City

Oxford

State/Province

Oxfordshire

ZIP/Postal Code

OX3 7LJ

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

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.

IPD Sharing Access Criteria

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.

IPD Sharing URL

https://vivli.org/ourmember/takeda/

Learn more about this trial

Recombinant Von Willebrand Factor in Subjects With Severe Von Willebrand Disease Undergoing Surgery

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