A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
NCT ID: NCT02932618
Last Updated: 2025-04-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
31 participants
INTERVENTIONAL
2017-11-06
2026-03-31
Brief Summary
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The participants will be treated with vonicog alfa 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.
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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On-demand Treatment
Participants will receive vonicog alfa (recombinant von Willebrand factor \[rVWF\]) treatment for non-surgical bleeding episodes over a 12 to 18-month period.
Vonicog alfa
Lyophilized powder and solvent to prepare solution for injection.
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.
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.
Vonicog alfa
Lyophilized powder and solvent to prepare solution for injection.
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.
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.
Vonicog alfa
Lyophilized powder and solvent to prepare solution for injection.
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.
Interventions
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Vonicog alfa
Lyophilized powder and solvent to prepare solution for injection.
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 Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
* Unable to tolerate are inadequately responsive to, or not a good candidate for 1-deamino-8-D-arginine vasopressin (DDAVP). Examples of participants who are not good candidates for DDAVP include participants with type 2B or type 3 VWD.
* 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
* 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 vonicog alfa 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 vonicog alfa or investigational device during the course of this study.
* Participant's legal representative is a family member or employee of the Investigator.
17 Years
ALL
No
Sponsors
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Takeda Development Center Americas, Inc.
INDUSTRY
Baxalta now part of Shire
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
Takeda
Locations
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University of Colorado Hemophilia & Thrombosis Center
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Florida College of Medicine
Jacksonville, Florida, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
St. Jude Affiliate Clinic at Novant Health
Charlotte, North Carolina, United States
Comprehensive Cancer Center of Wake Forest Unversity
Winston-Salem, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Rainbow Babies and Children's Hospital
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Texas Children's Cancer and Hematology Center
Houston, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
Comprehensive Center for Bleeding Disorders
Milwaukee, Wisconsin, United States
Medizinische Universität Innsbruck
Innsbruck, , Austria
AKH - Medizinische Universität Wien
Vienna, , Austria
UZ Leuven
Leuven, , Belgium
Fakultni nemocnice Brno
Brno, , Czechia
Hôpital Morvan
Brest, Finistere, France
Groupe Hospitalier Pellegrin - Hôpital Pellegrin
Bordeaux, , France
Groupement Hospitalier Est- Hôpital Louis Pradel
Bron, , France
CHU CAEN - Hôpital de la Côte de Nacre
Caen, , France
Groupement Hospitalier Sud - Hôpital Bicêtre
Le Kremlin-Bicêtre, , France
Hopital Cardiologique - CHU Lille
Lille, , France
CHU de Nantes Site Hotel Dieu
Nantes, , France
Hôpital Necker - Enfants Malades
Paris, , France
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, , Germany
Werlhof-Institut GmbH
Hanover, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Azienda Ospedaliera Universitaria Careggi
Florence, , Italy
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, , Italy
Azienda Ospedaliera Pediatrica Santobono Pausillipon
Napoli, , Italy
Ospedale Pediatrico Bambino Gesù
Roma, , Italy
Erasmus Medisch Centrum
Rotterdam, , Netherlands
SBEI HPE Altai State Medical University of MoH and SD
Barnaul, , Russia
SAIH "Kemerovo Regional Clinical Hospital"
Kemerovo, , Russia
FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA
Kirov, , Russia
Hospital General Universitario de Alicante
Alicante, , Spain
Hospital Universitari i Politecnic La Fe
Valencia, , Spain
Istanbul University Cerrahpasa Medical Faculty
Istanbul, , Turkey (Türkiye)
Ege University Medical Faculty
Izmir, , Turkey (Türkiye)
Ondokuz Mayis Univ. Med. Fac.
Samsun, , Turkey (Türkiye)
SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
Lviv, , Ukraine
Royal Manchester Children's Hospital
Manchester, Greater Manchester, United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Related Links
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To obtain more information on the study, click here/on this link
Other Identifiers
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2016-001477-33
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-509769-18-00
Identifier Type: CTIS
Identifier Source: secondary_id
071102
Identifier Type: -
Identifier Source: org_study_id
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