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RECRUITING
NCT02932618
PHASE3

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

View on ClinicalTrials.gov

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

Interventions

BIOLOGICAL

Vonicog alfa

Lyophilized powder and solvent to prepare solution for injection.

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.

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