New hope for kids with rare bleeding disorder: drug aims to stop bleeds before they start

NCT ID NCT05582993

First seen Nov 01, 2025

Summary

This study tests a drug called vonicog alfa to prevent bleeding in 24 children with severe von Willebrand disease, a condition that causes easy bruising and heavy bleeding. Participants receive the drug regularly for 12 months to see if it reduces bleeding episodes compared to their past experiences. The goal is to improve daily life by controlling the disease with ongoing treatment.

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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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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Azienda Ospedaliera Pediatrica Santobono Pausillipon

    ACTIVE_NOT_RECRUITING

    Naples, 80123, Italy

  • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino

    ACTIVE_NOT_RECRUITING

    Turin (Torino), 10126, Italy

  • Bleeding and Clotting Disorders Institute

    RECRUITING

    Peoria, Illinois, 61614, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's Health Care d/b/a Children's Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55404, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Children's Health Ireland

    RECRUITING

    Dublin, D12N512, Ireland

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Childrens Hospital of Michigan

    RECRUITING

    Detroit, Michigan, 48201, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Cure 4 the Kids

    RECRUITING

    Las Vegas, Nevada, 89135, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children NHS Foundation Trust

    RECRUITING

    London, WC1N 3HR, United Kingdom

    Contact

    Contact Email: •••••@•••••

  • Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419

    RECRUITING

    Lille, 59037, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Hopital Edouard Herriot - CHU Lyon

    RECRUITING

    Lyon, 69677, France

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Japanese Red Cross Narita Hospital

    RECRUITING

    Narita, Chiba, 286-8523, Japan

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Medical University of South Carolina (MUSC)

    RECRUITING

    Charleston, South Carolina, 29425, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Michigan State University Center for Bleeding Disorders & Clotting Disorders

    RECRUITING

    East Lansing, Michigan, 48824, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • NHO Beppu Medical Center

    RECRUITING

    Beppu, Oita Prefecture, 874-0011, Japan

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Nagasaki University Hospital

    RECRUITING

    Nagasaki, 852-8501, Japan

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Nara Medical University Hospital

    RECRUITING

    Kashihara, Nara, 634-8522, Japan

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • New York - Presbyterian/Weill Cornell Medical Center

    RECRUITING

    New York, New York, 10021, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Riley Hospital for Children Indiana University Health

    RECRUITING

    Indianapolis, Indiana, 46202, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Rutgers - Robert Wood Johnson Medical School

    RECRUITING

    New Brunswick, New Jersey, 08901, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35223, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University of Iowa Hospitals & Clinics PARENT

    RECRUITING

    Iowa City, Iowa, 52242, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

The condition(s) this trial relates to.

von Willebrand disease (hereditary or acquired)

As listed by the trial registrant

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