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A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)

Primary Purpose

Von Willebrand Disease (VWD)

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
rVWF
rFVIII
Sponsored by
Baxalta now part of Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Von Willebrand Disease (VWD)

Eligibility Criteria

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

Inclusion Criteria:

The participant will not be considered eligible for the study without meeting all of the criteria below.

Participants who have completed Study 071301 or Study 071102 (or participants who have completed the surgery arm treatment in Study 071102 and want to continue to receive on-demand (OD) treatment) and are willing to immediately transition into this study, must meet the following 2 criteria to be eligible for this study:

  • If female of childbearing potential, has a negative blood/urine pregnancy test at screening and agrees to employ highly effective birth control measures for the duration of the study.
  • Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.

New participants (Cohort 4) who meet the above 2 and ALL the following additional criteria are eligible for this study:

- Participant has a documented diagnosis of severe von Willebrand disease (VWD) (baseline von Willebrand factor: Ristocetin cofactor (VWF:RCo) <20 International Units per deciliter [IU/dL]) with a history of requiring substitution therapy with von Willebrand factor (vWF) concentrate to control bleeding:

  • Type 1 (VWF:RCo <20 IU/dL) or,
  • Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype), Type 2M or,
  • Type 3 (Von Willebrand factor antigen (VWF:Ag) less than or equal to (<=) 3 IU/dL).

Diagnosis is confirmed by genetic testing and multimer analysis, documented in participant history or at screening.

  • Participant has been receiving OD therapy with VWF products for at least 12 months, and prophylactic treatment is recommended by the investigator.
  • Participant has greater than or equal to (>=) 3 documented spontaneous bleeds (not including menorrhagia) requiring VWF treatment during the past 12 months.
  • Participant has available records that reliably evaluate type, frequency, and treatment of bleeding episodes for at least 12 months preceding enrollment; up to 24 months of retrospective data should be collected if available.
  • Participant is >=12 years old at the time of screening and has a body mass index >=15 but <40 kilogram per meter square (kg/m^2).

Exclusion Criteria:

The participant will be excluded from the study if any of the following exclusion criteria are met.

  • The participant has been diagnosed with Type 2N VWD, pseudo VWD, or another hereditary or acquired coagulation disorder other than VWD (eg, qualitative and quantitative platelet disorders or elevated prothrombin time (PT)/international normalized ratio [INR] >1.4).
  • The participant has a history or presence of a VWF inhibitor at screening.
  • 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 >=0.6 BU (by Bethesda assay).
  • The participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.
  • The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies.
  • The participant has a medical history of a thromboembolic event.
  • The participant is human immunodeficiency virus (HIV) positive with an absolute Helper T cell (CD4) count <200/cubic millimeters (mm^3).
  • 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, but not limited to any of the following: serum alanine aminotransferase (ALT) greater than 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-Pugh class B or C.
  • The participant has been diagnosed with renal disease, with a serum creatinine (CR) level >=2.5 milligrams per deciliter (mg/dL).
  • The participant has a platelet count <100,000/milliliter (mL) at screening.
  • The participant has been treated with an immunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to signing the informed consent (or assent, if appropriate).
  • The participant is pregnant or lactating at the time of enrollment.
  • The participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
  • 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.
  • The participant has a progressive fatal disease and/or life expectancy of less than 15 months.
  • For new OD participants, the participant is scheduled for a surgical intervention.
  • The participant is identified by the investigator as being unable or unwilling to cooperate with study procedures.
  • 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.
  • 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.

Delay criteria Only for Cohort 4, if the participant presents with an acute bleeding episodes or acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, and non-seasonal asthma) the screening visit will be postponed until the participant has recovered. For all other participants, end of study (EOS) visit for 071102 or 071301 will be completed per protocol and the completed EOS in Study 071102 or 071301 will also serve as the screening visit for this continuation study (SHP677-304).

Sites / Locations

  • Arkansas Children's Hospital Research InstituteRecruiting
  • University of Colorado HealthRecruiting
  • University of Florida College of MedicineRecruiting
  • Indiana Hemophilia and Thrombosis CenterRecruiting
  • Cincinnati Children's Hospital Medical CenterRecruiting
  • Rainbow Babies and Children's HospitalRecruiting
  • Nationwide Children's HospitalRecruiting
  • Medical University of South Carolina (MUSC)Recruiting
  • AKH - Medizinische Universität WienRecruiting
  • Hopital Cardiologique - CHU LilleRecruiting
  • Hôpital Necker - Enfants MaladesRecruiting
  • Groupement Hospitalier Est- Hôpital Louis PradelRecruiting
  • Groupe Hospitalier Pellegrin - Hôpital Pellegrin
  • Groupement Hospitalier Sud - Hôpital BicêtreRecruiting
  • Klinikum der Johann Wolfgang Goethe-UniversitaetRecruiting
  • Werlhof-Institut GmbHRecruiting
  • Azienda Ospedaliera Universitaria CareggiRecruiting
  • Fondazione IRCCS CA' Granda Ospedale Maggiore PoliclinicoRecruiting
  • Azienda Ospedaliera Pediatrica Santobono PausilliponRecruiting
  • Fondazione Policlinico Universitario Agostino Gemelli IRCCSRecruiting
  • Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La SapienzaRecruiting
  • Ospedale Pediatrico Bambino GesùRecruiting
  • Erasmus Medisch CentrumRecruiting
  • Erasmus Medisch CentrumRecruiting
  • SAIH "Kemerovo Regional Clinical Hospital"
  • FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA
  • Hospital General Universitario de AlicanteRecruiting
  • Hospital Universitario La PazRecruiting
  • Hospital Universitari i Politecnic La FeRecruiting
  • Istanbul University Oncology InstituteRecruiting
  • Ege University Medical FacultyRecruiting
  • Ege University Medical FacultyRecruiting
  • Ondokuz Mayis Univ. Med. Fac.Recruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

On-Demand

Prophylaxis

Arm Description

Participants will receive recombinant von Willebrand factor (rVWF) (with or without ADVATE).

Participants will receive recombinant von Willebrand factor (rVWF).

Outcomes

Primary Outcome Measures

Spontaneous Annualized Bleeding Rate (ABR)
Spontaneous ABR during prophylaxis treatment with rVWF (vonicog alfa)

Secondary Outcome Measures

Adverse Events (AEs)/Serious Adverse Events (SAEs)
AEs/ SAEs: incidence, severity, causality
Thromboembolic Events
Occurrence of thromboembolic events
Hypersensitivity Reactions
Occurrence of hypersensitivity reactions
Number of Participants who Develop Neutralizing Antibodies to von Willebrand factor (VWF)
Number of participants who develop neutralizing antibodies (inhibitors) to VWF
Number of Participants who Develop Neutralizing Antibodies to Factor VIII (FVIII)
Number of participants who develop neutralizing antibodies (inhibitors) to FVIII
Number of Participants who Develop Total Binding Antibodies to von Willebrand factor (VWF)
Number of participants who develop total binding antibodies to VWF
Number of Participants who Develop Total Binding Antibodies to Factor VIII (FVIII)
Number of participants who develop total binding antibodies to FVIII
Number of Participants who Develop Binding Antibodies to Chinese hamster ovary (CHO) proteins
Number of participants who develop binding antibodies to CHO proteins
Number of Participants who Develop Binding Antibodies to Mouse Immunoglobulin G (IgG)
Number of participants who develop binding antibodies to mouse IgG
Number of Participants who Develop Binding Antibodies to recombinant Furin (rFurin)
Number of participants who develop binding antibodies to rFurin
Number of Participants With Clinically Significant Changes in Vital Signs
Clinically significant changes in vital signs (body temperature, blood pressure, respiration and pulse) will be assessed from baseline up to 3 years
Number of Participants With Clinically Significant Changes in Laboratory Parameters
Clinically significant changes in laboratory parameters (clinical chemistry, fasting lipid panel and determination of fat-soluble vitamins, bile acids and other cholestasis biochemical markers) will be assessed from baseline up to 3 years
Spontaneous Annualized Bleeding Rate (ABR) Under Prophylactic Treatment
Spontaneous ABR under prophylactic treatment with rVWF (vonicog alfa) while enrolled in the study
Categorized Weekly Number of Infusions
Categorized as 1, 2 or >=3 during prophylactic treatment with rVWF (vonicog alfa)
Categorized Spontaneous Annualized Bleeding Rate (ABR)
Categorized as 0, 1-2, 3-5, or >5 bleeding episodes during rVWF (vonicog alfa) prophylaxis
Time to First Bleeding Event
Under each prophylaxis regimen
Spontaneous Annualized Bleeding Rate (ABR) by Location of Bleeding
Gastrointestinal [GI], epistaxis, joint bleeding, menorrhagia, oral, muscle and soft tissue, etc. while on prophylactic treatment with rVWF (vonicog alfa)
Total Number of Infusions
Total number of infusions during prophylactic treatment with rVWF (vonicog alfa)
Average Number of Infusions
Per week during prophylactic treatment with rVWF (vonicog alfa)
Total Weight Adjusted Consumption of recombinant von Willebrand factor (rVWF) (vonicog alfa)
During prophylactic treatment
Number of Participants who Achieve Transfusion-free Maintenance of Hemoglobin Levels
Transfusion free maintenance of hemoglobin levels over time
Ferritin Levels Over Time
Ferritin levels over time will be reported.
Overall Hemostatic Efficacy Rating
At the resolution of bleed with respect to the treatment of bleeding episodes for the initial 12 months of study
Number of Infusions
Number of infusions of rVWF (vonicog alfa) and ADVATE (rFVIII, octocog alfa) utilized to treat bleeding episodes while enrolled in the study
Weight-adjusted Consumption
Weight-adjusted consumption of rVWF (vonicog alfa) and ADVATE (rFVIII, octocog alfa) per bleeding episode while enrolled in the study

Full Information

First Posted
December 20, 2018
Last Updated
August 10, 2023
Sponsor
Baxalta now part of Shire
Collaborators
Takeda Development Center Americas, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03879135
Brief Title
A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
Official Title
A Phase 3b, Prospective, Open-Label, Uncontrolled, Multicenter Study on Long-Term Safety and Efficacy of rVWF in Pediatric and Adult Subjects With Severe Von Willebrand Disease (VWD)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 1, 2019 (Actual)
Primary Completion Date
December 31, 2023 (Anticipated)
Study Completion Date
December 31, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Baxalta now part of Shire
Collaborators
Takeda Development Center Americas, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants during the first 12 months on study treatment. The participants will be treated with rVWF for a maximum of 3 years. Their von Willebrand Disease will be treated according to Investigational product (IP) dosing directions.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Von Willebrand Disease (VWD)

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
71 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
On-Demand
Arm Type
Experimental
Arm Description
Participants will receive recombinant von Willebrand factor (rVWF) (with or without ADVATE).
Arm Title
Prophylaxis
Arm Type
Experimental
Arm Description
Participants will receive recombinant von Willebrand factor (rVWF).
Intervention Type
Biological
Intervention Name(s)
rVWF
Other Intervention Name(s)
Vonvendi, Vonicog alfa
Intervention Description
Recombinant von Willebrand factor
Intervention Type
Biological
Intervention Name(s)
rFVIII
Other Intervention Name(s)
Octocog alfa, ADVATE
Intervention Description
Recombinant Factor VIII
Primary Outcome Measure Information:
Title
Spontaneous Annualized Bleeding Rate (ABR)
Description
Spontaneous ABR during prophylaxis treatment with rVWF (vonicog alfa)
Time Frame
First 12 months of treatment
Secondary Outcome Measure Information:
Title
Adverse Events (AEs)/Serious Adverse Events (SAEs)
Description
AEs/ SAEs: incidence, severity, causality
Time Frame
Throughout the study participation period, up to 3 years
Title
Thromboembolic Events
Description
Occurrence of thromboembolic events
Time Frame
Throughout the study participation period, up to 3 years
Title
Hypersensitivity Reactions
Description
Occurrence of hypersensitivity reactions
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants who Develop Neutralizing Antibodies to von Willebrand factor (VWF)
Description
Number of participants who develop neutralizing antibodies (inhibitors) to VWF
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants who Develop Neutralizing Antibodies to Factor VIII (FVIII)
Description
Number of participants who develop neutralizing antibodies (inhibitors) to FVIII
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants who Develop Total Binding Antibodies to von Willebrand factor (VWF)
Description
Number of participants who develop total binding antibodies to VWF
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants who Develop Total Binding Antibodies to Factor VIII (FVIII)
Description
Number of participants who develop total binding antibodies to FVIII
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants who Develop Binding Antibodies to Chinese hamster ovary (CHO) proteins
Description
Number of participants who develop binding antibodies to CHO proteins
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants who Develop Binding Antibodies to Mouse Immunoglobulin G (IgG)
Description
Number of participants who develop binding antibodies to mouse IgG
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants who Develop Binding Antibodies to recombinant Furin (rFurin)
Description
Number of participants who develop binding antibodies to rFurin
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants With Clinically Significant Changes in Vital Signs
Description
Clinically significant changes in vital signs (body temperature, blood pressure, respiration and pulse) will be assessed from baseline up to 3 years
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants With Clinically Significant Changes in Laboratory Parameters
Description
Clinically significant changes in laboratory parameters (clinical chemistry, fasting lipid panel and determination of fat-soluble vitamins, bile acids and other cholestasis biochemical markers) will be assessed from baseline up to 3 years
Time Frame
Throughout the study participation period, up to 3 years
Title
Spontaneous Annualized Bleeding Rate (ABR) Under Prophylactic Treatment
Description
Spontaneous ABR under prophylactic treatment with rVWF (vonicog alfa) while enrolled in the study
Time Frame
Throughout the study participation period, up to 3 years
Title
Categorized Weekly Number of Infusions
Description
Categorized as 1, 2 or >=3 during prophylactic treatment with rVWF (vonicog alfa)
Time Frame
Throughout the study participation period, up to 3 years
Title
Categorized Spontaneous Annualized Bleeding Rate (ABR)
Description
Categorized as 0, 1-2, 3-5, or >5 bleeding episodes during rVWF (vonicog alfa) prophylaxis
Time Frame
Throughout the study participation period, up to 3 years
Title
Time to First Bleeding Event
Description
Under each prophylaxis regimen
Time Frame
Throughout the study participation period, up to 3 years
Title
Spontaneous Annualized Bleeding Rate (ABR) by Location of Bleeding
Description
Gastrointestinal [GI], epistaxis, joint bleeding, menorrhagia, oral, muscle and soft tissue, etc. while on prophylactic treatment with rVWF (vonicog alfa)
Time Frame
Throughout the study participation period, up to 3 years
Title
Total Number of Infusions
Description
Total number of infusions during prophylactic treatment with rVWF (vonicog alfa)
Time Frame
Throughout the study participation period, up to 3 years
Title
Average Number of Infusions
Description
Per week during prophylactic treatment with rVWF (vonicog alfa)
Time Frame
Throughout the study participation period, up to 3 years
Title
Total Weight Adjusted Consumption of recombinant von Willebrand factor (rVWF) (vonicog alfa)
Description
During prophylactic treatment
Time Frame
Throughout the study participation period, up to 3 years
Title
Number of Participants who Achieve Transfusion-free Maintenance of Hemoglobin Levels
Description
Transfusion free maintenance of hemoglobin levels over time
Time Frame
Throughout the study participation period, up to 3 years
Title
Ferritin Levels Over Time
Description
Ferritin levels over time will be reported.
Time Frame
Throughout the study participation period, up to 3 years
Title
Overall Hemostatic Efficacy Rating
Description
At the resolution of bleed with respect to the treatment of bleeding episodes for the initial 12 months of study
Time Frame
Initial 12 months of study
Title
Number of Infusions
Description
Number of infusions of rVWF (vonicog alfa) and ADVATE (rFVIII, octocog alfa) utilized to treat bleeding episodes while enrolled in the study
Time Frame
Throughout the study participation period, up to 3 years
Title
Weight-adjusted Consumption
Description
Weight-adjusted consumption of rVWF (vonicog alfa) and ADVATE (rFVIII, octocog alfa) per bleeding episode while enrolled in the study
Time Frame
Throughout the study participation period, up to 3 years

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: The participant will not be considered eligible for the study without meeting all of the criteria below. Participants who have completed Study 071301 or Study 071102 (or participants who have completed the surgery arm treatment in Study 071102 and want to continue to receive on-demand (OD) treatment) and are willing to immediately transition into this study, must meet the following 2 criteria to be eligible for this study: If female of childbearing potential, has a negative blood/urine pregnancy test at screening and agrees to employ highly effective birth control measures for the duration of the study. Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol. New participants (Cohort 4) who meet the above 2 and ALL the following additional criteria are eligible for this study: - Participant has a documented diagnosis of severe von Willebrand disease (VWD) (baseline von Willebrand factor: Ristocetin cofactor (VWF:RCo) <20 International Units per deciliter [IU/dL]) with a history of requiring substitution therapy with von Willebrand factor (vWF) concentrate to control bleeding: Type 1 (VWF:RCo <20 IU/dL) or, Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype), Type 2M or, Type 3 (Von Willebrand factor antigen (VWF:Ag) less than or equal to (<=) 3 IU/dL). Diagnosis is confirmed by genetic testing and multimer analysis, documented in participant history or at screening. Participant has been receiving OD therapy with VWF products for at least 12 months, and prophylactic treatment is recommended by the investigator. Participant has greater than or equal to (>=) 3 documented spontaneous bleeds (not including menorrhagia) requiring VWF treatment during the past 12 months. Participant has available records that reliably evaluate type, frequency, and treatment of bleeding episodes for at least 12 months preceding enrollment; up to 24 months of retrospective data should be collected if available. Participant is >=12 years old at the time of screening and has a body mass index >=15 but <40 kilogram per meter square (kg/m^2). Exclusion Criteria: The participant will be excluded from the study if any of the following exclusion criteria are met. The participant has been diagnosed with Type 2N VWD, pseudo VWD, or another hereditary or acquired coagulation disorder other than VWD (eg, qualitative and quantitative platelet disorders or elevated prothrombin time (PT)/international normalized ratio [INR] >1.4). The participant has a history or presence of a VWF inhibitor at screening. 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 >=0.6 BU (by Bethesda assay). The participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins. The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies. The participant has a medical history of a thromboembolic event. The participant is human immunodeficiency virus (HIV) positive with an absolute Helper T cell (CD4) count <200/cubic millimeters (mm^3). 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, but not limited to any of the following: serum alanine aminotransferase (ALT) greater than 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-Pugh class B or C. The participant has been diagnosed with renal disease, with a serum creatinine (CR) level >=2.5 milligrams per deciliter (mg/dL). The participant has a platelet count <100,000/milliliter (mL) at screening. The participant has been treated with an immunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to signing the informed consent (or assent, if appropriate). The participant is pregnant or lactating at the time of enrollment. The participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia). 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. The participant has a progressive fatal disease and/or life expectancy of less than 15 months. For new OD participants, the participant is scheduled for a surgical intervention. The participant is identified by the investigator as being unable or unwilling to cooperate with study procedures. 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. 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. Delay criteria Only for Cohort 4, if the participant presents with an acute bleeding episodes or acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, and non-seasonal asthma) the screening visit will be postponed until the participant has recovered. For all other participants, end of study (EOS) visit for 071102 or 071301 will be completed per protocol and the completed EOS in Study 071102 or 071301 will also serve as the screening visit for this continuation study (SHP677-304).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Shire Contact
Phone
+1 866 842 5335
Email
ClinicalTransparency@takeda.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Arkansas Children's Hospital Research Institute
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
stinekimoc@uams.edu
First Name & Middle Initial & Last Name & Degree
Stine Kimo
Facility Name
University of Colorado Health
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
michael.wang@ucdenver.edu
First Name & Middle Initial & Last Name & Degree
Michael Wang, MD
Facility Name
University of Florida College of Medicine
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
twynn@ufl.edu
First Name & Middle Initial & Last Name & Degree
Wynn Tung
Facility Name
Indiana Hemophilia and Thrombosis Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site contact
Email
ashapiro@ihtc.org
First Name & Middle Initial & Last Name & Degree
Amy Shapiro, MD
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Phone
513-803-4617
Email
Eric.mullins@cchmc.org
First Name & Middle Initial & Last Name & Degree
Mullins Eric, MD
Facility Name
Rainbow Babies and Children's Hospital
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
sanjay.ahuja@uhhospitals.org
First Name & Middle Initial & Last Name & Degree
Ahuja Sanjay
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
amy.dunn@nationwidechildrens.org
First Name & Middle Initial & Last Name & Degree
Dunn Amy
Facility Name
Medical University of South Carolina (MUSC)
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
bergmans@musc.edu
First Name & Middle Initial & Last Name & Degree
Bergmann Shayla
Facility Name
AKH - Medizinische Universität Wien
City
Vienna
Country
Austria
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
christoph.male@meduniwien.ac.at
First Name & Middle Initial & Last Name & Degree
Male Christoph
Facility Name
Hopital Cardiologique - CHU Lille
City
Lille Cedex
State/Province
Nord
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
sophie.susen@chru-lille.fr
First Name & Middle Initial & Last Name & Degree
Susen Sophie
Facility Name
Hôpital Necker - Enfants Malades
City
Paris cedex 15
State/Province
Paris
ZIP/Postal Code
75015
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
annie.harroche@aphp.fr
First Name & Middle Initial & Last Name & Degree
Harroche-Angel Annie
Facility Name
Groupement Hospitalier Est- Hôpital Louis Pradel
City
Bron cedex
ZIP/Postal Code
69677
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
anne.lienhart@chu-lyon.fr
First Name & Middle Initial & Last Name & Degree
Lienhart Anne
Facility Name
Groupe Hospitalier Pellegrin - Hôpital Pellegrin
City
Gironde
Country
France
Individual Site Status
Completed
Facility Name
Groupement Hospitalier Sud - Hôpital Bicêtre
City
Le Kremlin Bicêtre cedex
ZIP/Postal Code
94270
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
roseline.doiron@aphp.fr
First Name & Middle Initial & Last Name & Degree
D oiron Roseline
Facility Name
Klinikum der Johann Wolfgang Goethe-Universitaet
City
Frankfurt
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
wolfgang.miesbach@kgu.de
First Name & Middle Initial & Last Name & Degree
Miesbach Wolfgang
Facility Name
Werlhof-Institut GmbH
City
Hannover
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
mail@werlhof-institut.de
First Name & Middle Initial & Last Name & Degree
von Depka Prondzinski Mario
Facility Name
Azienda Ospedaliera Universitaria Careggi
City
Firenze
ZIP/Postal Code
50134
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
castaman@aou-careggi.toscana.it
First Name & Middle Initial & Last Name & Degree
Giancarlo Castaman, MD
Facility Name
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
City
Milano
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
flora.peyvandi@policlinico.mi.it
First Name & Middle Initial & Last Name & Degree
Peyvandi Flora
Facility Name
Azienda Ospedaliera Pediatrica Santobono Pausillipon
City
Napoli
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
mischiavulli@gmail.com
First Name & Middle Initial & Last Name & Degree
Schiavulli Michele
Facility Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
City
Roma
ZIP/Postal Code
00168
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
raimondo.decristofaro@unicatt.it
First Name & Middle Initial & Last Name & Degree
De Cristofaro Raimondo
Facility Name
Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza
City
Roma
ZIP/Postal Code
00185
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
chistolini@bce.uniroma1.it
First Name & Middle Initial & Last Name & Degree
Chistolini Antonio
Facility Name
Ospedale Pediatrico Bambino Gesù
City
Roma
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
matteo.luciani@opbg.net
First Name & Middle Initial & Last Name & Degree
Luciani Matteo
Facility Name
Erasmus Medisch Centrum
City
Rotterdam
ZIP/Postal Code
3015 AA
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site contact
Email
f.leebeek@erasmusmc.nl
First Name & Middle Initial & Last Name & Degree
Franciscus Leebeek, MD, Ph.D.
Facility Name
Erasmus Medisch Centrum
City
Rotterdam
ZIP/Postal Code
3015 CN
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site contact
Email
m.cnossen@erasmusmc.nl
First Name & Middle Initial & Last Name & Degree
Marjon Cnossen, MD, Ph.D.
Facility Name
SAIH "Kemerovo Regional Clinical Hospital"
City
Kemerovo
ZIP/Postal Code
650066
Country
Russian Federation
Individual Site Status
Completed
Facility Name
FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA
City
Kirov
ZIP/Postal Code
610017
Country
Russian Federation
Individual Site Status
Completed
Facility Name
Hospital General Universitario de Alicante
City
Alicante
ZIP/Postal Code
03010
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
marco_pas@gva.es
First Name & Middle Initial & Last Name & Degree
Pascual Marco Vera, MD
Facility Name
Hospital Universitario La Paz
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
monicamsalces@gmail.com
First Name & Middle Initial & Last Name & Degree
Monica Martin Salces, MD
Facility Name
Hospital Universitari i Politecnic La Fe
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site contact
Email
cid_ana@gva.es
First Name & Middle Initial & Last Name & Degree
Ana Rosa Cid Haro, MD
Facility Name
Istanbul University Oncology Institute
City
Istanbul
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
bulent.zulfikar@istanbulmedicare.com
First Name & Middle Initial & Last Name & Degree
Zulfikar Osman Bulent
Facility Name
Ege University Medical Faculty
City
Izmir
ZIP/Postal Code
35040
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site contact
Email
drfahrisahin@gmail.com
First Name & Middle Initial & Last Name & Degree
Fahri Sahin, MD
Facility Name
Ege University Medical Faculty
City
Izmir
ZIP/Postal Code
35100
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
drcanbalkan@gmail.com
First Name & Middle Initial & Last Name & Degree
Can Balkan, MD
Facility Name
Ondokuz Mayis Univ. Med. Fac.
City
Samsun
ZIP/Postal Code
55139
Country
Turkey
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site Contact
Email
can68ucar@yahoo.com.tr
First Name & Middle Initial & Last Name & Degree
Canan Albayrak, MD

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/
Links:
URL
https://clinicaltrials.takeda.com/study-detail/5f6b5fe84db2bf003ab47b19
Description
To obtain more information on the study, click here/on this link

Learn more about this trial

A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)

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