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Von Willebrand Disease in the Netherlands (WiN-Pro)

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ClinicalTrials.gov Identifier: NCT03521583
Recruitment Status : Not yet recruiting
First Posted : May 10, 2018
Last Update Posted : May 23, 2018
Sponsor:
Collaborators:
Stichting Haemophilia (Dutch Haemophilia Foundation)
CSL Behring
Shire
Information provided by (Responsible Party):
Frank W.G. Leebeek, MD, PhD, Erasmus Medical Center

Brief Summary:
The primary aim of this study is to prospectively investigate the current bleeding tendency of children and adults with VWD.

Condition or disease
Von Willebrand Disease

Detailed Description:

Von Willebrand disease (VWD) is the most common inherited bleeding disorder, and is characterized by a defective platelet adhesion and aggregation. VWD is caused by a reduced (type 1), an abnormal function (type 2) or a complete absence (type 3) of von Willebrand factor (VWF).

In recent years, large retrospective cohort studies have provided valuable insights on the clinical presentation, bleeding phenotype, quality of life, diagnostics, genetics and treatment of patients with VWD. One of these large studies is the von Willebrand in the Netherlands (WiN) study, which is a nationwide cross sectional study of moderate and severe von Willebrand disease patients, that was initiated in 2007. Over 800 VWD patients were included in the WiN study, which was about 80% of all known VWD patients in the Netherlands. Although the WiN study and large retrospective studies in other countries provided important insights in understanding VWD, some significant challenges remain and large prospective studies are lacking to provide answers.

All large retrospective cohort studies have assessed the bleeding phenotype of patients with VWD using bleeding scores or retrospective questionnaires. Bleeding scores calculate the sum of all bleeding episodes during lifetime. Therefore, they provide useful information on the bleeding tendency during lifetime. However, bleeding scores do not provide information on the change of bleeding tendency. If a patient had a period in his or her lifetime in which he or she had many bleeding episodes, then the bleeding score is high. Though, the patient could have had those bleeds 30 years ago and did not have a bleeding episode since then. Therefore, bleeding scores do not provide information on the current bleeding phenotype of VWD patients. Furthermore, previous studies provided limited information on the frequency of mild bleedings, like gum bleeding or epistaxis, that occur in daily life but do not require therapy. Nevertheless, these bleeding episodes can cause a major impairment in quality of life. This is especially important in children, because school-going children with VWD have a lower quality of life and have a different bleeding tendency, characterized by more cutaneous bleeding (81%), oropharyngeal bleeding (64%) and epistaxis (56%).

Therefore, the primary aim of this study is to prospectively investigate the current bleeding tendency of children and adults with VWD.


Study Type : Observational
Estimated Enrollment : 1100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Von Willebrand Disease in the Netherlands - Prospective Study (WiN-Pro)
Estimated Study Start Date : October 2018
Estimated Primary Completion Date : January 2021
Estimated Study Completion Date : January 2021

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. Bleeding rate [ Time Frame: 2 years ]
    Number of bleedings in an individual divided by the follow-up duration



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Von Willebrand disease patients known in a Hemophilia Treatment Center in the Netherlands
Criteria

Inclusion Criteria:

  • Historically lowest VWF:Ag and/or VWF:RCo and/or VWF:CB ≤ 0.30 IU/mL and/or FVIII:C ≤ 0.40 IU/mL
  • Treatment at a Hemophilia treatment center in the Netherlands
  • All types of VWD
  • All ages

Exclusion Criteria:

- Other known bleeding disorders present.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03521583


Contacts
Contact: Frank WG Leebeek, MD, PhD +31107031369 f.leebeek@erasmusmc.nl

Locations
Netherlands
Academic Medical Center Not yet recruiting
Amsterdam, Netherlands
Haga Hospital Not yet recruiting
Den Haag, Netherlands
Maxima Medical Center
Eindhoven, Netherlands
University Medical Center Groningen Not yet recruiting
Groningen, Netherlands
Leiden University Medical Center Not yet recruiting
Leiden, Netherlands
Maastricht University Medical Center + Not yet recruiting
Maastricht, Netherlands
Radboud University Medical Center Not yet recruiting
Nijmegen, Netherlands
Erasmus University Medical Center Not yet recruiting
Rotterdam, Netherlands
University Medical Center Utrecht Not yet recruiting
Utrecht, Netherlands
Sponsors and Collaborators
Erasmus Medical Center
Stichting Haemophilia (Dutch Haemophilia Foundation)
CSL Behring
Shire
Investigators
Principal Investigator: Frank WG Leebeek, MD, PhD Erasmus Medical Center
Study Director: Ferdows Atiq, MD Erasmus Medical Center
Study Chair: Marjon H Cnossen, MD, PhD Erasmus Medical Center
Study Chair: Karin Fijnvandraat, MD, PhD Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Study Chair: Jeroen Eikenboom, MD, PhD Leiden University Medical Center
Study Chair: Johanna G van der Bom, PhD Leiden University Medical Center
Study Chair: Britta AP Laros-van Gorkom, MD, PhD Radboud University
Study Chair: Karina Meijer, MD, PhD University Medical Center Groningen
Study Chair: Karin PM van Galen, MD, PhD UMC Utrecht
Study Chair: Joke de Meris Netherlands Hemophilia Society

Publications:

Responsible Party: Frank W.G. Leebeek, MD, PhD, MD, PhD, Erasmus Medical Center
ClinicalTrials.gov Identifier: NCT03521583     History of Changes
Other Study ID Numbers: NL62238.078.18
First Posted: May 10, 2018    Key Record Dates
Last Update Posted: May 23, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Frank W.G. Leebeek, MD, PhD, Erasmus Medical Center:
von Willebrand disease
von Willebrand factor
Nationwide study
Prospective study

Additional relevant MeSH terms:
Von Willebrand Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn