New hope for kids with rare bleeding disorder: study tests rVWF treatment

NCT ID NCT02932618

First seen May 16, 2026 · Last updated May 16, 2026

Summary

This study tested a medicine called vonicog alfa (rVWF) to control bleeding in 47 children under 18 with severe von Willebrand disease, a genetic bleeding disorder. The treatment was given with or without another factor product (ADVATE) for 12-18 months. The goal was to see how well it stops bleeding episodes and if it is safe, but it is not a cure—children will still need ongoing management.

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Contacts and locations

Locations

  • AKH - Medizinische Universität Wien

    Vienna, 1090, Austria

  • Azienda Ospedaliera Pediatrica Santobono Pausillipon

    Naples, 80122, Italy

  • Azienda Ospedaliera Universitaria Careggi

    Florence, 50134, Italy

  • Bleeding and Clotting Disorders Institute

    Peoria, Illinois, 61615, United States

  • CHU CAEN - Hôpital de la Côte de Nacre

    Caen, 14033, France

  • CHU de Nantes Site Hotel Dieu

    Nantes, 44093, France

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Comprehensive Cancer Center of Wake Forest Unversity

    Winston-Salem, North Carolina, 27157, United States

  • Comprehensive Center for Bleeding Disorders

    Milwaukee, Wisconsin, 53225, United States

  • Ege University Medical Faculty

    Izmir, 35100, Turkey (Türkiye)

  • Erasmus Medisch Centrum

    Rotterdam, 3015 CN, Netherlands

  • FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA

    Kirov, 610027, Russia

  • Fakultni nemocnice Brno

    Brno, 613 00, Czechia

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Groupe Hospitalier Pellegrin - Hôpital Pellegrin

    Bordeaux, 33000, France

  • Groupement Hospitalier Est- Hôpital Louis Pradel

    Bron, 69677, France

  • Groupement Hospitalier Sud - Hôpital Bicêtre

    Le Kremlin-Bicêtre, 94270, France

  • Hopital Cardiologique - CHU Lille

    Lille, 59037, France

  • Hospital General Universitario de Alicante

    Alicante, 03010, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026, Spain

  • Hôpital Morvan

    Brest, Finistere, 29609, France

  • Hôpital Necker - Enfants Malades

    Paris, 75743, France

  • Indiana Hemophilia and Thrombosis Center

    Indianapolis, Indiana, 46260, United States

  • Istanbul University Cerrahpasa Medical Faculty

    Istanbul, 34098, Turkey (Türkiye)

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Medizinische Hochschule Hannover

    Hanover, 30625, Germany

  • Medizinische Universität Innsbruck

    Innsbruck, 6020, Austria

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Ondokuz Mayis Univ. Med. Fac.

    Samsun, 55139, Turkey (Türkiye)

  • Ospedale Pediatrico Bambino Gesù

    Roma, 00165, Italy

  • Rainbow Babies and Children's Hospital

    Cleveland, Ohio, 44106, United States

  • Royal Manchester Children's Hospital

    Manchester, Greater Manchester, M13 9WL, United Kingdom

  • SAIH "Kemerovo Regional Clinical Hospital"

    Kemerovo, 650066, Russia

  • SBEI HPE Altai State Medical University of MoH and SD

    Barnaul, 656038, Russia

  • SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

    Lviv, 79044, Ukraine

  • St. Jude Affiliate Clinic at Novant Health

    Charlotte, North Carolina, 28204, United States

  • Texas Children's Cancer and Hematology Center

    Houston, Texas, 77030, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • UZ Leuven

    Leuven, 3000, Belgium

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • University of Colorado Hemophilia & Thrombosis Center

    Aurora, Colorado, 80045, United States

  • University of Florida College of Medicine

    Jacksonville, Florida, 32610, United States

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198, United States

  • Werlhof-Institut GmbH

    Hanover, 30159, Germany

Conditions

Explore the condition pages connected to this study.