A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

August 10, 2023 updated by: Baxalta now part of Shire

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease

The main aim of the study is to check effectiveness, side effects, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric participants (less than (<)18 years of age) with severe hereditary von Willebrand disease (VWD).

The participants will be treated with rVWF for 12-18 months. Their von Willebrand Disease will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be followed up at clinics or over telephone calls.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

34

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Austria
      • Innsbruck, Austria, 6020
        • Completed
        • Medizinische Universität Innsbruck
      • Vienna, Austria, 1090
        • Recruiting
        • AKH - Medizinische Universitat Wien
        • Principal Investigator:
          • Christoph Male, MD, MSc
        • Contact:
  • Belgium
      • Leuven, Belgium, 3000
  • Czechia
      • Brno, Czechia, 613 00
        • Completed
        • Fakultní Nemocnice Brno
  • France
      • Bordeaux Cedex, France, 33000
        • Recruiting
        • Groupe Hospitalier Pellegrin - Hôpital Pellegrin
        • Principal Investigator:
          • Yoann Huguenin, MD
        • Contact:
      • Bron cedex, France, 69677
        • Recruiting
        • Groupement Hospitalier Est- Hôpital Louis Pradel
        • Contact:
        • Principal Investigator:
          • Lucia Rugeri, MD
      • Caen cedex 9, France, 14033
        • Recruiting
        • CHU CAEN - Hôpital de la Côte de Nacre
        • Contact:
        • Principal Investigator:
          • Annie Borel-Derlon, MD
      • Le Kremlin-Bicêtre, France, 94270
        • Recruiting
        • Groupement Hospitalier Sud - Hôpital Bicêtre
        • Principal Investigator:
          • Roseline D oiron, MD
        • Contact:
      • Lille Cedex, France, 59037
        • Recruiting
        • Hopital Cardiologique - CHU Lille
        • Principal Investigator:
          • Sophie Susen, MD, MSc, PhD
        • Contact:
      • Nantes Cedex 1, France, 44093
        • Completed
        • CHU de Nantes Site Hotel Dieu
      • Paris cedex 15, France, 75743
        • Recruiting
        • Hopital Necker - Enfants Malades
        • Principal Investigator:
          • Annie Harroche-Angel, MD
        • Contact:
    • Finistere
      • Brest, Finistere, France, 29609
        • Completed
        • Hopital Morvan
  • Germany
      • Hamburg, Germany, 20246
        • Completed
        • Universitaetsklinikum Hamburg-Eppendorf
      • Hannover, Germany, 30625
        • Completed
        • Medizinische Hochschule Hannover
      • Hannover, Germany, 30159
        • Completed
        • Werlhof-Institut GmbH
  • Italy
      • Firenze, Italy, 50134
        • Recruiting
        • Azienda Ospedaliera Universitaria Careggi
        • Contact:
        • Principal Investigator:
          • Giancarlo Castaman, MD
      • Milano, Italy, 20122
        • Recruiting
        • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
        • Contact:
        • Principal Investigator:
          • Flora Peyvandi, MD
      • Napoli, Italy, 80122
        • Recruiting
        • Azienda Ospedaliera Pediatrica Santobono Pausillipon
        • Principal Investigator:
          • Michele Schiavulli, MD
        • Contact:
      • Roma, Italy, 00165
        • Recruiting
        • Ospedale Pediatrico Bambino Gesù
        • Contact:
        • Principal Investigator:
          • Matteo Luciani, MD
  • Netherlands
      • Rotterdam, Netherlands, 3015 CN
        • Completed
        • Erasmus Medisch Centrum
  • Russian Federation
      • Barnaul, Russian Federation, 656038
        • Completed
        • SBEI HPE Altai State Medical University of MoH and SD
      • Kemerovo, Russian Federation, 650066
        • Completed
        • SAIH "Kemerovo Regional Clinical Hospital"
      • Kirov, Russian Federation, 610027
        • Active, not recruiting
        • FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA
  • Spain
      • Alicante, Spain, 03010
        • Recruiting
        • Hospital General Universitario de Alicante
        • Contact:
        • Principal Investigator:
          • Pascual Marco Vera, MD
      • Valencia, Spain, 46026
        • Recruiting
        • Hospital Universitari i Politècnic La Fe
        • Contact:
        • Principal Investigator:
          • Ana Rosa Cid Haro, MD
  • Turkey
      • Istanbul, Turkey, 34098
      • Izmir, Turkey, 35100
        • Recruiting
        • Ege University Medical Faculty
        • Contact:
        • Principal Investigator:
          • Ramazan Kavakli
      • Samsun, Turkey, 55139
        • Recruiting
        • Ondokuz Mayis Univ. Med. Fac.
        • Contact:
        • Principal Investigator:
          • Canan Albayrak, MD
  • Ukraine
      • Lviv, Ukraine, 79044
        • Completed
        • SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
  • United Kingdom
    • Greater Manchester
      • Manchester, Greater Manchester, United Kingdom, M13 9WL
        • Completed
        • Royal Manchester Children's Hospital
  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado Hemophilia & Thrombosis Center
        • Contact:
        • Principal Investigator:
          • Michael Wang, MD
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Completed
        • Children's National Medical Center
    • Florida
      • Jacksonville, Florida, United States, 32610
        • Recruiting
        • University of Florida College of Medicine
        • Contact:
        • Principal Investigator:
          • Tung Wynn, MD
    • Illinois
      • Peoria, Illinois, United States, 61615
        • Recruiting
        • Bleeding and Clotting Disorders Institute
        • Contact:
        • Principal Investigator:
          • Jonathan Roberts, MD
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Completed
        • Indiana Hemophilia and Thrombosis Center
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • Completed
        • University of Nebraska Medical Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Completed
        • St. Jude Affiliate Clinic at Novant Health
      • Winston-Salem, North Carolina, United States, 27157
        • Completed
        • Comprehensive Cancer Center of Wake Forest Unversity
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Contact:
        • Principal Investigator:
          • Eric Mullins, MD
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • Rainbow Babies and Children's Hospital
        • Principal Investigator:
          • Sanjay Ahuja, MD, MSc, MBA
        • Contact:
      • Columbus, Ohio, United States, 43205
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Completed
        • Children's Hospital of Philadelphia
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Medical University of South Carolina
        • Principal Investigator:
          • Shayla Bergmann, MD
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Texas Children's Hospital
        • Principal Investigator:
          • Rosa Diaz
      • Houston, Texas, United States, 77030
        • Recruiting
        • Texas Children's Cancer and Hematology Center
        • Contact:
        • Principal Investigator:
          • Lakshmi Srivaths, MD
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53225
        • Completed
        • Comprehensive Center for Bleeding Disorders

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 17 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor [VWF:RCo] less than [<] 20 percent [%]):

    • Type 1 (VWF:RCo <20 International Units per deciliter [IU/dL]); or
    • Type 2A (VWF:RCo <20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (Factor VIII coagulation activity [FVIII:C] <10 % and historically documented genetics), Type 2M; or
    • Type 3 (VWF:Ag less than or equal to [=<] 3 IU/dL).
  • Age 0 to <18 years at the time of Screening.
  • The participant has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent.
  • If female of childbearing potential, participant presents with a negative serum pregnancy test.
  • If applicable, participant agrees to employ adequate birth control measures for the duration of the study.
  • The participant and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, which should also be confirmed based on a pre-screening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the participants compliance with the study requirements.

Additional inclusion criteria for both previously treated participants and participants undergoing surgery are as follows:

  • Unable to tolerate or are inadequately responsive to deamino-delta-D-arginine vasopressin (DDAVP).
  • The participant has had a minimum of 1 documented bleed requiring VWF coagulation factor replacement therapy (i.e. treatment with a VWF product) during the previous 12 months prior to enrollment and overall historically 3 or more exposure days (EDs) to VWF replacement therapy.

Additional inclusion criterion for previously untreated participants are as follows:

- The participant has not received prior VWF coagulation factor replacement therapy.

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] greater than [>] 1.4).
  • History or presence of a VWF inhibitor at Screening.
  • History or presence of a Factor VIII (FVIII) inhibitor with a titer greater than or equal [>=] 0.4 Bethesda units (BU) (by Nijmegen assay) or >=0.6 BU (by Bethesda assay).
  • Documented history of a VWF: RCo half-life <6 hours.
  • Known hypersensitivity to any of the components of the study drug, such as mouse or hamster proteins.
  • Medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis/asthma, food allergies, or animal allergies.
  • Medical history of a thromboembolic event.
  • Human immunodeficiency virus (HIV) positive, with an absolute CD4 count <200/ cubic millimeter (mm^3).
  • In the judgment of the Investigator, the participant has another clinically significant concomitant disease (e.g. uncontrolled hypertension, cancer) that may pose additional risks for the participant.
  • Diagnosis of significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) of 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (e.g. 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 >=2.5 milligram per deciliter (mg/dL).
  • Immunomodulatory drug treatment other than anti-retroviral chemotherapy (e.g. α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 milligram per day [mg/day] (excluding topical treatment [e.g. ointments, nasal sprays]), within 30 days prior to signing the informed consent (or assent, if appropriate).
  • If female, participant is pregnant or lactating at the time informed consent (or assent, if appropriate) 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 other than rVWF or investigational device during the course of this study.
  • Participant's legal representative is a family member or employee of the Investigator.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: On-demand Treatment
Participants will receive recombinant von Willebrand factor (rVWF) treatment for non-surgical bleeding episodes over a 12 to 18-month period.
Biological: von Willebrand factor (Recombinant)
Lyophilized powder and solvent to prepare solution for injection.
Other Names:
  • BAX 111
  • rVWF
  • VONVENDI
  • BAX111
Biological: Antihemophilic Factor (Recombinant)
Packaged in single boxes with 2 glass vials, with one vial containing the lyophilized ADVATE and the second vial containing the diluent.
Other Names:
  • ADVATE
  • rFVIII
  • Recombinant Factor VIII
Experimental: Elective Surgery
12-24 hours prior to surgery and within 3 hours of surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing.
Biological: von Willebrand factor (Recombinant)
Lyophilized powder and solvent to prepare solution for injection.
Other Names:
  • BAX 111
  • rVWF
  • VONVENDI
  • BAX111
Biological: Antihemophilic Factor (Recombinant)
Packaged in single boxes with 2 glass vials, with one vial containing the lyophilized ADVATE and the second vial containing the diluent.
Other Names:
  • ADVATE
  • rFVIII
  • Recombinant Factor VIII
Experimental: Emergency Surgery
Within 3 hours prior to surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing.
Biological: von Willebrand factor (Recombinant)
Lyophilized powder and solvent to prepare solution for injection.
Other Names:
  • BAX 111
  • rVWF
  • VONVENDI
  • BAX111
Biological: Antihemophilic Factor (Recombinant)
Packaged in single boxes with 2 glass vials, with one vial containing the lyophilized ADVATE and the second vial containing the diluent.
Other Names:
  • ADVATE
  • rFVIII
  • Recombinant Factor VIII

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Hemostatic Efficacy
Time Frame: Within 24 hours after the last infusion of study drug following the onset of the bleeding episode (if/when the severity and/or duration of the bleeding requires the infusion of the study drug)
Treatment success for rVWF-treated nonsurgical bleeding episodes (using a 4-point scale: Excellent, Good, Moderate, None).
Within 24 hours after the last infusion of study drug following the onset of the bleeding episode (if/when the severity and/or duration of the bleeding requires the infusion of the study drug)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Treated Nonsurgical Bleeding Episodes With an Efficacy Rating of 'Excellent' or 'Good'
Time Frame: Throughout the study duration of approximately 6.5 years
If/when the severity and/or duration of the bleeding requires the infusion of the study drug.
Throughout the study duration of approximately 6.5 years
Number of Infusions per Bleeding Episode
Time Frame: Throughout the study duration of approximately 6.5 years
Throughout the study duration of approximately 6.5 years
Number of Recombinant Von Willebrand Factor (rVWF) Units per Bleeding Episode
Time Frame: Throughout the study duration of approximately 6.5 years
Throughout the study duration of approximately 6.5 years
Number of ADVATE Units (if needed) per Bleeding Episode
Time Frame: Throughout the study duration of approximately 6.5 years
Throughout the study duration of approximately 6.5 years
Elective or Emergency Surgery: Assessment of Hemostatic Efficacy - Immediately After Surgery
Time Frame: Immediately after surgery
Assessed by the operating surgeon, based on a 4-point ordinal scale: Excellent, Good, Moderate, None.
Immediately after surgery
Elective or Emergency Surgery: Overall Assessment of Hemostatic Efficacy 24 Hours After the Last Perioperative Infusion of rVWF
Time Frame: 24 hours after last perioperative rVWF infusion
Assessed by the Investigator (hematologist) on a 4-point ordinal scale: Excellent, Good, Moderate, None.
24 hours after last perioperative rVWF infusion
Elective or Emergency surgery: Overall Assessment of Hemostatic Efficacy Day 7 Post-operative
Time Frame: Post-operative Day 7
Assessed by the Investigator (hematologist) on a 4-point ordinal scale: Excellent, Good, Moderate, None.
Post-operative Day 7
Elective or Emergency surgery: Overall Assessment of Hemostatic Efficacy Day 14 Post-operative
Time Frame: Post-operative Day 14
Assessed by the Investigator (hematologist) on a 4-point ordinal scale: Excellent, Good, Moderate, None.
Post-operative Day 14
Incidence and Severity of Adverse Events (AEs)
Time Frame: Throughout the study period of approximately 6.5 years
Throughout the study period of approximately 6.5 years
Incidence of Thrombotic Events
Time Frame: Throughout the study period of approximately 6.5 years
Throughout the study period of approximately 6.5 years
Incidence of Severe Hypersensitivity Reactions
Time Frame: Throughout the study period of approximately 6.5 years
Throughout the study period of approximately 6.5 years
Development of Neutralizing Antibodies to von Willebrand Factor (VWF) and Factor VIII (FVIII)
Time Frame: Throughout the study period of approximately 6.5 years
Throughout the study period of approximately 6.5 years
Development of Total Binding Antibodies to von Willebrand Factor (VWF)
Time Frame: Throughout the study period of approximately 6.5 years
Throughout the study period of approximately 6.5 years
Development of Antibodies to Chinese Hamster Ovary (CHO) Proteins, Murine Immunoglobulin G (IgG), and rFurin
Time Frame: Throughout the study period of approximately 6.5 years
Throughout the study period of approximately 6.5 years
Area Under the Plasma Concentration/Time Curve From 0 to 96 Hours Post-Infusion (AUC0-96h) for Von Willebrand factor: ristocetin cofactor (VWF:Rco), von Willebrand factor: antigen (VWF:Ag) and von Willebrand factor: collagen binding capacity (VWF:CB)
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-inf) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Mean Residence Time (MRT) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Time to Reach Maximal Plasma Concentration (Tmax) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Maximal Plasma Concentration (Cmax) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Clearance (CL) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Incremental Recovery (IR) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
In-vivo Recovery (IVR) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Elimination Phase Half-life (T1/2) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Volume of Distribution at Steady State (Vss) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Area Under the Plasma Concentration/Time Curve From 0 to 96 Hours Post-infusion (AUC0-96h) for Factor VIII (FVIII) Activity
Time Frame: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours
Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Baxalta now part of Shire

Collaborators

Takeda Development Center Americas, Inc.

Investigators

  • Study Director: Study Director, Takeda

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 18, 2017

Primary Completion (Estimated)

March 31, 2024

Study Completion (Estimated)

March 31, 2024

Study Registration Dates

First Submitted

October 12, 2016

First Submitted That Met QC Criteria

October 12, 2016

First Posted (Estimated)

October 13, 2016

Study Record Updates

Last Update Posted (Actual)

August 14, 2023

Last Update Submitted That Met QC Criteria

August 10, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 071102
  • 2016-001477-33 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD 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 Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Von Willebrand Disease

Clinical Trials on von Willebrand factor (Recombinant)

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Cote d'Ivoire

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe