Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

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

NCT ID NCT03879135

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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.

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

38 people

The number who actually took part.

Started

Apr 2019

Finished

Jan 2025

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

0 years and older

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: The participant will not be considered eligible for the study without meeting all of the criteria below. Participants who have completed Study 071301 or Study 071102 (or participants who have completed the surgery arm treatment in Study 071102 and want to continue to receive on-demand (OD) treatment) and are willing to immediately transition into this study, must meet the following 2 criteria to be eligible for this study: * If female of childbearing potential, has a negative blood/urine pregnancy test at screening and agrees to employ highly effective birth control measures for the duration of the study. * Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol. New participants (Cohort 4) who meet the above 2 and ALL the following additional criteria are eligible for this study: \- Participant has a documented diagnosis of severe von Willebrand disease (VWD) (baseline von Willebrand factor: Ristocetin cofactor (VWF:RCo) \<20 International Units per deciliter \[IU/dL\]) with a history of requiring substitution therapy with von Willebrand factor (vWF) concentrate to control bleeding: * Type 1 (VWF:RCo \<20 IU/dL) or, * Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype), Type 2M or, * Type 3 (Von Willebrand factor antigen (VWF:Ag) less than or equal to (\<=) 3 IU/dL). Diagnosis is confirmed by genetic testing and multimer analysis, documented in participant history or at screening. * Participant has been receiving OD therapy with VWF products for at least 12 months, and prophylactic treatment is recommended by the investigator. * Participant has greater than or equal to (\>=) 3 documented spontaneous bleeds (not including menorrhagia) requiring VWF treatment during the past 12 months. * Participant has available records that reliably evaluate type, frequency, and treatment of bleeding episodes for at least 12 months preceding enrollment; up to 24 months of retrospective data should be collected if available. * Participant is \>=12 years old at the time of screening and has a body mass index \>=15 but \<40 kilogram per meter square (kg/m\^2). Exclusion Criteria: The participant will be excluded from the study if any of the following exclusion criteria are met. * The participant has been diagnosed with Type 2N VWD, pseudo VWD, or another hereditary or acquired coagulation disorder other than VWD (eg, qualitative and quantitative platelet disorders or elevated prothrombin time (PT)/international normalized ratio \[INR\] \>1.4). * The participant has a history or presence of a VWF inhibitor at screening. * The participant has a history or presence of a Factor VIII (FVIII) inhibitor with a titer \>=0.4 Bethesda units (BU) (by Nijmegen modified Bethesda assay) or \>=0.6 BU (by Bethesda assay). * The participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins. * The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies. * The participant has a medical history of a thromboembolic event. * The participant is human immunodeficiency virus (HIV) positive with an absolute Helper T cell (CD4) count \<200/cubic millimeters (mm\^3). * The participant has been diagnosed with significant liver disease per investigator's medical assessment of the participant's current condition or medical history or as evidenced by, but not limited to any of the following: serum alanine aminotransferase (ALT) greater than 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child-Pugh class B or C. * The participant has been diagnosed with renal disease, with a serum creatinine (CR) level \>=2.5 milligrams per deciliter (mg/dL). * The participant has a platelet count \<100,000/milliliter (mL) at screening. * The participant has been treated with an immunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to signing the informed consent (or assent, if appropriate). * The participant is pregnant or lactating at the time of enrollment. * The participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia). * The participant has participated in another clinical study involving another investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study. * The participant has a progressive fatal disease and/or life expectancy of less than 15 months. * For new OD participants, the participant is scheduled for a surgical intervention. * The participant is identified by the investigator as being unable or unwilling to cooperate with study procedures. * The participant has a mental condition rendering him/her unable to understand the nature, scope and possible consequences of the study and/or evidence of an uncooperative attitude. * The participant is member of the study team or in a dependent relationship with one of the study team members which includes close relatives (i.e., children, partner/spouse, siblings and parents) as well as employees. Delay criteria Only for Cohort 4, if the participant presents with an acute bleeding episodes or acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, and non-seasonal asthma) the screening visit will be postponed until the participant has recovered. For all other participants, end of study (EOS) visit for 071102 or 071301 will be completed per protocol and the completed EOS in Study 071102 or 071301 will also serve as the screening visit for this continuation study (SHP677-304).

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Severe von Willebrand disease are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

More trials for these conditions

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