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New hope for rare bleeding disorder: Long-Term drug shows promise

NCT ID NCT03879135

Completed Disease control Sponsor: Baxalta now part of Shire Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study tested a drug called rVWF (Vonvendi) in 38 children and adults with severe von Willebrand disease, a bleeding disorder. The goal was to see if regular use of the drug could reduce the number of spontaneous bleeding episodes over a year. Participants received treatment for up to three years, and researchers tracked safety and bleeding rates.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Recombinant von Willebrand factor (rVWF, also called Vonvendi or Vonicog alfa)

What this could lead to

If successful, this could provide a long-term treatment option to prevent spontaneous bleeding episodes in people with severe von Willebrand disease.

What could go wrong

This is a small, open-label study with no control group, so results may not apply to all patients. Side effects or lack of effectiveness in some individuals remain possible.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

hereditary von Willebrand disease von Willebrand disease (hereditary or acquired)

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

VON WILLEBRAND DISEASE (VWD)

Contacts and locations

Locations

  • AKH - Medizinische Universität Wien

    Vienna, Austria

  • Arkansas Children's Hospital Research Institute

    Little Rock, Arkansas, 72202, United States

  • Azienda Ospedaliera Pediatrica Santobono Pausillipon

    Naples, Italy

  • Azienda Ospedaliera Universitaria Careggi

    Florence, 50134, Italy

  • Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza

    Roma, 00185, Italy

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Ege University Medical Faculty

    Izmir, 35040, Turkey (Türkiye)

  • Ege University Medical Faculty

    Izmir, 35100, Turkey (Türkiye)

  • Erasmus Medisch Centrum

    Rotterdam, 3015 AA, Netherlands

  • Erasmus Medisch Centrum

    Rotterdam, 3015 CN, Netherlands

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

    Kirov, 610017, Russia

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milan, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 00168, Italy

  • Groupe Hospitalier Pellegrin - Hôpital Pellegrin

    Gironde, 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, Nord, France

  • Hospital General Universitario de Alicante

    Alicante, 03010, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hôpital Necker - Enfants Malades

    Paris, Paris, 75015, France

  • Indiana Hemophilia and Thrombosis Center

    Indianapolis, Indiana, 46260, United States

  • Istanbul University Oncology Institute

    Istanbul, Turkey (Türkiye)

  • Klinikum der Johann Wolfgang Goethe-Universitaet

    Frankfurt, Germany

  • Medical University of South Carolina (MUSC)

    Charleston, South Carolina, 29425, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Ondokuz Mayis Univ. Med. Fac.

    Samsun, 55139, Turkey (Türkiye)

  • Ospedale Pediatrico Bambino Gesù

    Roma, Italy

  • Rainbow Babies and Children's Hospital

    Cleveland, Ohio, 44106, United States

  • SAIH "Kemerovo Regional Clinical Hospital"

    Kemerovo, 650066, Russia

  • University of Colorado Health

    Aurora, Colorado, 80045, United States

  • University of Florida College of Medicine

    Gainesville, Florida, 32610, United States

  • Werlhof-Institut GmbH

    Hanover, Germany