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A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
Sponsor: Baxalta now part of Shire
Summary
The main aim of the study is to check effectiveness, side effects, and tolerability of vonicog alfa (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 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.
Official title: 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
Key Details
Gender
All
Age Range
Any - 17 Years
Study Type
INTERVENTIONAL
Enrollment
31
Start Date
2017-11-06
Completion Date
2026-03-31
Last Updated
2025-04-30
Healthy Volunteers
No
Conditions
Interventions
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.
Locations (46)
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
Naples, 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