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New hope for kids with rare bleeding disease: study tests rVWF therapy

NCT ID NCT02932618

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a medicine called vonicog alfa (rVWF), sometimes given with another drug (ADVATE), to control bleeding in 47 children under 18 with severe von Willebrand disease. The treatment was given for 12-18 months, and doctors checked how well it stopped bleeding and if it caused side effects. The goal was to see if this approach is safe and effective for managing this lifelong bleeding disorder.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

47 people

The number who actually took part.

Started

Nov 2017

Finished

Apr 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor \[VWF:RCo\] less than \[\<\] 20 percent \[%\]): * Type 1 (VWF:RCo \<20 International Units per deciliter \[IU/dL\]); or * Type 2A (VWF:RCo \<20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (Factor VIII coagulation activity \[FVIII:C\] \<10 % and historically documented genetics), Type 2M; or * Type 3 (VWF:Ag less than or equal to \[=\<\] 3 IU/dL). * Age 0 to \<18 years at the time of Screening. * The participant has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent. * If female of childbearing potential, participant presents with a negative serum pregnancy test. * If applicable, participant agrees to employ adequate birth control measures for the duration of the study. * The participant and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, which should also be confirmed based on a pre-screening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the participants compliance with the study requirements. Additional inclusion criteria for both previously treated participants and participants undergoing surgery are as follows: * Unable to tolerate are inadequately responsive to, or not a good candidate for 1-deamino-8-D-arginine vasopressin (DDAVP). Examples of participants who are not good candidates for DDAVP include participants with type 2B or type 3 VWD. * The participant has had a minimum of 1 documented bleed requiring VWF coagulation factor replacement therapy (i.e. treatment with a VWF product) during the previous 12 months prior to enrollment and overall historically 3 or more exposure days (EDs) to VWF replacement therapy. Additional inclusion criterion for previously untreated participants are as follows: \- The participant has not received prior VWF coagulation factor replacement therapy. Exclusion Criteria: * Diagnosis of pseudo-VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time \[PT\]/international normalized ratio \[INR\] greater than \[\>\] 1.4). * History or presence of a VWF inhibitor at Screening. * History or presence of a Factor VIII (FVIII) inhibitor with a titer greater than or equal \[\>=\] 0.4 Bethesda units (BU) (by Nijmegen assay) or \>=0.6 BU (by Bethesda assay). * Documented history of a VWF: RCo half-life \<6 hours. * Known hypersensitivity to any of the components of the study drug, such as mouse or hamster proteins. * Medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis/asthma, food allergies, or animal allergies. * Medical history of a thromboembolic event. * Human immunodeficiency virus (HIV) positive, with an absolute CD4 count \<200/ cubic millimeter (mm\^3). * In the judgment of the Investigator, the participant has another clinically significant concomitant disease (e.g. uncontrolled hypertension, cancer) that may pose additional risks for the participant. * Diagnosis of significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) of 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child B or C. * Diagnosis of renal disease, with a serum creatinine level \>=2.5 milligram per deciliter (mg/dL). * Immunomodulatory drug treatment other than anti-retroviral chemotherapy (e.g. α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 milligram per day \[mg/day\] (excluding topical treatment \[e.g. ointments, nasal sprays\]), within 30 days prior to signing the informed consent (or assent, if appropriate). * If female, participant is pregnant or lactating at the time informed consent (or assent, if appropriate) is obtained. * Participant has participated in another clinical study involving an investigational product (IP), other than vonicog alfa with or without ADVATE, or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP other than vonicog alfa or investigational device during the course of this study. * Participant's legal representative is a family member or employee of the Investigator.

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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

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.